Latest news with #NoonanSyndrome
Globe and Mail
3 days ago
- Business
- Globe and Mail
Noonan Syndrome Market Growth to Accelerate in Forecast Period (2023-2032), DelveInsight Analyzes
DelveInsight's 'Noonan Syndrome Market Insights, Epidemiology, and Market Forecast-2032″ report offers an in-depth understanding of the Noonan Syndrome, historical and forecasted epidemiology as well as the Noonan Syndrome market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan. The latest healthcare forecast report provides an in-depth analysis of Noonan Syndrome, offering comprehensive insights into the Noonan Syndrome revenue trends, prevalence, and treatment landscape. The report delves into key Noonan Syndrome statistics, highlighting the current and projected market size, while examining the efficacy and development of emerging Noonan Syndrome therapies. Additionally, we cover the landscape of Noonan Syndrome clinical trials, providing an overview of ongoing and upcoming studies that are poised to shape the future of Noonan Syndrome treatment. This report is an essential resource for understanding the market dynamics and the evolving therapeutic options within the Noonan Syndrome space. Some of the key facts of the Noonan Syndrome Market Report: The Noonan Syndrome market size is anticipated to grow with a significant CAGR during the study period (2019-2032) In May 2025, Novo Nordisk's once-weekly Sogroya (somapacitan) demonstrated non-inferiority compared to the once-daily growth hormone Norditropin (somatropin) in a Phase III trial focused on pediatric growth disorders. The Danish company reported that Sogroya enhanced the annual growth rate in pre-pubertal children born small for gestational age (SGA), those with Noonan syndrome (NS), or those with idiopathic short stature (ISS), as shown in the Phase III REAL8 basket study (NCT05330325). Key Noonan Syndrome Companies: Sanofi, Novo Nordisk A/S, BioMarin Pharmaceutical, and others Key Noonan Syndrome Therapies: MAXOMAT ®, somatropin, Vosoritide Injection, and others The Noonan Syndrome market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Noonan Syndrome pipeline products will significantly revolutionize the Noonan Syndrome market dynamics. The prevalence of Noonan Syndrome Occurs in approximately 1 in 1,000 to 2,500 live births worldwide Noonan Syndrome is One of the more common genetic syndromes affecting multiple systems Noonan Syndrome Affects males and females equally, with no significant gender bias Caused by mutations in genes involved in the RAS/MAPK pathway, including PTPN11, SOS1, RAF1, and others About 50% of cases are due to mutations in the PTPN11 gene Noonan Syndrome Overview Noonan Syndrome is a genetic disorder that affects multiple parts of the body. It is characterized by distinctive facial features, short stature, heart defects (such as pulmonary valve stenosis), developmental delays, and other health issues. The condition is caused by mutations in genes involved in the RAS/MAPK signaling pathway, which plays a key role in cell growth and development. Noonan Syndrome occurs in both males and females and varies widely in severity. Early diagnosis and management can help address the associated complications and improve quality of life. Get a Free sample for the Noonan Syndrome Market Forecast, Size & Share Analysis Report: Noonan Syndrome Epidemiology The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends. Noonan Syndrome Epidemiology Segmentation: The Noonan Syndrome market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into: Total Prevalence of Noonan Syndrome Prevalent Cases of Noonan Syndrome by severity Gender-specific Prevalence of Noonan Syndrome Diagnosed Cases of Episodic and Chronic Noonan Syndrome Download the report to understand which factors are driving Noonan Syndrome epidemiology trends @ Noonan Syndrome Epidemiology Forecast Noonan Syndrome Drugs Uptake and Pipeline Development Activities The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Noonan Syndrome market or expected to get launched during the study period. The analysis covers Noonan Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share. The report also covers the Noonan Syndrome Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies. Noonan Syndrome Therapies and Key Companies Discover more about therapies set to grab major Noonan Syndrome market share @ Noonan Syndrome Treatment Landscape Noonan Syndrome Market Drivers Rising Diagnosis Rates Advancements in Genetic Research Pharmaceutical Collaborations Government Initiatives Noonan Syndrome Market Barriers Lack of Approved Therapies High Treatment Costs Limited Awareness Genetic Heterogeneity Scope of the Noonan Syndrome Market Report Study Period: 2019–2032 Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan] Key Noonan Syndrome Companies: Sanofi, Novo Nordisk A/S, BioMarin Pharmaceutical, and others Key Noonan Syndrome Therapies: MAXOMAT ®, somatropin, Vosoritide Injection, and others Noonan Syndrome Therapeutic Assessment: Noonan Syndrome current marketed and Noonan Syndrome emerging therapies Noonan Syndrome Market Dynamics: Noonan Syndrome market drivers and Noonan Syndrome market barriers Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies Noonan Syndrome Unmet Needs, KOL's views, Analyst's views, Noonan Syndrome Market Access and Reimbursement Table of Contents 1. Noonan Syndrome Market Report Introduction 2. Executive Summary for Noonan Syndrome 3. SWOT analysis of Noonan Syndrome 4. Noonan Syndrome Patient Share (%) Overview at a Glance 5. Noonan Syndrome Market Overview at a Glance 6. Noonan Syndrome Disease Background and Overview 7. Noonan Syndrome Epidemiology and Patient Population 8. Country-Specific Patient Population of Noonan Syndrome 9. Noonan Syndrome Current Treatment and Medical Practices 10. Noonan Syndrome Unmet Needs 11. Noonan Syndrome Emerging Therapies 12. Noonan Syndrome Market Outlook 13. Country-Wise Noonan Syndrome Market Analysis (2019–2032) 14. Noonan Syndrome Market Access and Reimbursement of Therapies 15. Noonan Syndrome Market Drivers 16. Noonan Syndrome Market Barriers 17. Noonan Syndrome Appendix 18. Noonan Syndrome Report Methodology 19. DelveInsight Capabilities 20. Disclaimer 21. About DelveInsight About DelveInsight DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Gaurav Bora Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: NV Country: United States Website:
Irish Examiner
22-05-2025
- Health
- Irish Examiner
Lorraine Desmond on raising children with CFC: 'I couldn't have predicted the joy my two boys would bring'
A Tuesday in December 2011, we had an appointment in CUMH with a professor of genetics from Dublin. December 13 — the day our two- and three-year-old sons Alex and Rian were diagnosed with CFC (cardiofaciocutaneous) syndrome. I'd heard of CFC. The boys had been very unwell babies, with lots of feeding and growth issues. When you put symptoms into Google it throws back a selection of possibilities. When you're focused on a topic, talk to other parents with similar issues, people come into your life, other mums, American mums especially. They'd have said different syndromes so I'd heard of CFC, but I didn't expect it. Professor Green took bloods. He took photos of the boys. He pointed out differences in their hands. I thought: 'Even their hands are different' — their little hands that were perfect to us were a feature of this syndrome. He said, based on their features, hands, and head shape, they had CFC. He was 110% certain, but there had never been two in the one family before. The expectation would be for just one in a family, unless either parent had it — we didn't. That's why he took bloods. CFC is part of a family of syndromes. Another example is Noonan Syndrome, which is lower on the scale of disability. I was disappointed to hear CFC. Although the boys were so young, they weren't where they should be. I knew now there was no hope they'd get to where they should be because there'd be an intellectual disability. Of course, we'd had our suspicions, but you don't ever want those suspicions confirmed. Because then it's true, it's real life. Until the point of confirmation, you feel you can make it better. So you sit in this moment and you feel, OK, this is it now — you feel there's a full stop, a full stop for hope, that you can make it better. You're starting the next paragraph then, figuratively. Two children with a rare disease... The statistics for CFC syndrome are 1 in 810,000. The odds increase dramatically for having two. Lorraine Desmond with her boys Alex and Rian. Picture: Dan Linehan We're the only family in the world with two children with CFC where neither parent has it. We knew then what we were dealing with; there were no more unknowns. It was time to get moving and make them the best they could be. The boys had no speech at this point; Rian was tube-fed, Alex was eating but very limited. They were failing to thrive, not meeting milestones. As a three-year-old Alex had lots of behavioural challenges, banging his head, hurting his brother because he couldn't tell us what was wrong. Rian's issues were more medical, including not sleeping. It was the same syndrome, different presentations. Those early years were so difficult, we wouldn't go back for all the tea in China. They attend Our Lady of Good Counsel Special School in Ballincollig. Once they got the support of a multidisciplinary team and amazing SNAs they started being able to talk, communicate their needs and wants, which reduced the behaviour difficulties. I remember the day Alex came home from school, looking for a biscuit in the cupboard. Before, he'd have had a tantrum because of not being able to make himself understood. Now he was able to make the sound and the Lámh sign for biscuit — it was incredible! You knew then there was hope — more speech would come, more sounds. It was the first glimpse there was something coming. Lorraine Desmond. Picture Dan Linehan Their fine and gross motor skills improved; they were able to put on their coats; feed themselves. I remember Rian sitting up in his high chair, a tiny little fellow, saying 'I do it' — he wanted to feed himself his own breakfast. For me not to have to feed him, that was a really emotional moment. They started to make friends. Their community was expanding, but so was ours. Alex today knows his own mind. We call him the detective. He asks question after question. If he meets someone, he'll have a good interpretation of them in five questions. Rian is equally social. He loves people and is really in tune with people's feelings. Looking back, that day of diagnosis was a positive moment, though we didn't think so then. It gave us an answer, helped us move forward. There was no more wondering. I couldn't have predicted then the level of joy they'd bring everybody around them. They joined the Marvel Club recently. People have been offering to host them in their homes for playdates — people seek out their company. We go through moments of grief. I'd always thought you grieve when they're diagnosed. But as they get older, there are still milestones, like Alex leaving school next year. There's grief that he won't be going to college like his older sister. There's anxiety — what happens when we get older; when they do? And guilt. Are we doing enough? But the joy... It's not that 'they're so cute'. No, they're really difficult; it's seeing people's interactions with them that makes us so happy. We're really proud they're out in the community and are liked by people. Our Lady of Good Counsel Special School, Ballincollig, is fundraising for a garden room, with a €41,000 target. The 'Bikers, Buns and Blooms 2025' motorbike run with family fun is this Sunday, May 25, at the school. The motorbike rally is run by Na Préacháin MCC. See: Our Lady of Good Counsel School on Facebook. Donate at Our Lady of Good Counsel at
Yahoo
12-05-2025
- Health
- Yahoo
Novo Nordisk A/S: Once-weekly Sogroya® (somapacitan) is an efficacious and well-tolerated long-acting growth hormone in children with growth disorders: results from REAL8 phase 3 basket study presented at the joint Congress of ESPE and ESE
The REAL8 trial showed that after 52 weeks, once-weekly Sogroya® (somapacitan) had similar clinical outcomes and safety profile to once-daily Norditropin® (somatropin) in children born small for gestational age (SGA)1, or with Noonan syndrome (NS)2, or with idiopathic short stature3. Superiority was achieved for once-weekly somapacitan versus daily growth hormone in children with NS2, as well as compared to lower doses of daily growth hormone in children born SGA1. These conditions are often associated with significant health challenges and a high treatment burden from daily injections4, which can lead to a lack of adherence and put successful treatment outcomes at risk. Novo Nordisk is committed to bringing our expertise and scientific innovation to help improve the lives of children with conditions that impact their growth. Bagsværd, Denmark, 12 May 2025 – Novo Nordisk today presented data from the phase 3 REAL8 basket study, which showed that once-weekly Sogroya® (somapacitan) was non-inferior to the once-daily growth hormone Norditropin® (somatropin) in improving yearly growth rate (as measured by height velocity [HV] at Week 52) in pre-pubertal children born small for gestational age (SGA)1, or with Noonan syndrome (NS)2, or with idiopathic short stature (ISS)3. In addition, superiority was achieved for once-weekly Sogroya® versus daily growth hormone in children with NS2, as well as compared to lower doses of daily growth hormone in children born SGA1. REAL8 data showed that Sogroya® was well-tolerated, with no safety or tolerability issues identified compared to once daily growth hormone1-3. Insulin-like growth factor 1 (IGF-1) response in patients treated with once-weekly Sogroya® was similar to those treated with daily growth hormone1-3. Results from the Turner syndrome (TS) sub-study of REAL8 will be available later this year. These data were presented as three late-breaking abstracts at the first Joint Congress of the European Society for Paediatric Endocrinology (ESPE) and European Society of Endocrinology (ESE) in Copenhagen, Denmark1-3. 'Children with growth failure face many health challenges beyond just being shorter than their peers. They often have metabolic disruptions and developmental difficulties that can seriously affect their wellbeing and quality of life, as well as long-term effects such as increased risk of cardiovascular disease or type 2 diabetes,' said Professor Agnès Linglart, Professor of Paediatrics at the Bicêtre Paris-Saclay University and Hospital, France, and one of the lead investigators on REAL8. 'The REAL8 data presented today marks an important step forward in providing these patients with an effective, once-weekly option that can potentially reduce treatment burden and improve adherence and treatment outcomes.' The REAL8 trial achieved its primary endpoints for the first three sub-studies, demonstrating that once-weekly Sogroya® was non-inferior to once-daily growth hormone treatment at Week 52 across the three indications presented: In children born SGA, Sogroya® demonstrated superior estimated mean HV when compared with a lower dose (0.035 mg/kg/day) of somatropin (11.0 vs 9.4 cm/year), and non-inferior estimated mean HV when compared with a higher dose (0.067 mg/kg/day) of somatropin (11.0 vs 11.1 cm/year)1. In children with NS, Sogroya® demonstrated superior estimated mean HV compared with somatropin (10.4 vs 9.2 cm/year)2. In children with ISS, Sogroya® demonstrated non-inferior estimated mean HV compared with daily somatropin (10.5 vs 10.5 cm/year)3. Non-adherence to growth hormone treatment is a common problem which puts successful treatment outcomes at risk. Daily injections can be a burden for children and their caregivers, leading to lack of adherence due to discomfort or pain at injection sites, inconvenience and disruption to daily life5. One study showed that missed daily injections resulted in a difference of 6.1 cm in height over 3 years when comparing nonadherent with adherent patients4. 'Treatment adherence is an issue when it comes to improving outcomes in children with growth failure. Imagine if a child misses only one day of treatment each week, amounting to 52 missed days per year. Over a seven-year treatment window, this results in one year of missed treatment and can have a significant knock-on impact on their health,' said Martin Lange, executive vice president for Development at Novo Nordisk. 'We are committed to providing a portfolio of growth hormone therapies with flexibility in administration timing and missed doses, which may better suit the needs of children with growth failure. These encouraging results from REAL8 mark a significant step forward in achieving that commitment.' In April 2025, based on the data from REAL8 and REAL9, the three indications (SGA, NS and ISS) were submitted for regulatory review in both the EU and US. About REAL8The REAL8 study is part of the ongoing REAL clinical trial programme, it is a randomised, open-label, active-controlled, parallel-group, phase 3 trial evaluating the efficacy and safety of once-weekly Sogroya® (somapacitan) in children born SGA, or with TS, NS or ISS. The primary treatment period was 52 weeks followed by a two-year safety extension phase6. REAL8 has an innovative basket trial design that is investigating once-weekly Sogroya® (somapacitan) in four different but related indications (SGA, TS, NS and ISS) under one trial protocol6. This trial design allows for a more efficient clinical development process by speeding up recruitment and consolidating resources7, potentially bringing treatment to patients with these conditions sooner. This is the first time a trial design of this type has been implemented in the growth disorder space. In REAL8, pre-pubertal children with NS, TS or ISS were randomised to receive either once-weekly Sogroya® (somapacitan) 0.24 mg/kg/week or once-daily somatropin 0.050 mg/kg/day; children born SGA were randomised to receive either somapacitan 0.24 mg/kg/week, or low dose of somatropin 0.035 mg/kg/day, or high dose of somatropin 0.067 mg/kg/day6. About REAL9The REAL9 study is part of the ongoing REAL clinical trial programme, it is a single-group assignment, phase 3 study evaluating the safety and efficacy of once-weekly Sogroya® (somapacitan) in children 10 years or older born SGA, or with TS, NS or ISS. The study will last for approximately 3 years8. About once-weekly Sogroya® Once-weekly Sogroya® (somapacitan) is a prescription human growth hormone analogue, similar to current daily growth hormone. It is currently approved for once-weekly treatment of children and adults who do not produce enough growth hormone9,10. Using albumin-binding prolongation technology, Sogroya® attaches to albumin, a protein in the blood, to help delay its removal from the body. This well-established technology has been used for over 20 years in Novo Nordisk's diabetes treatment. It allows for the growth hormone to work longer11. About Novo NordiskNovo Nordisk is a leading global healthcare company founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 77,400 people in 80 countries and markets its products in around 170 countries. For more information, visit Facebook, Instagram, X, LinkedIn and YouTube. Contacts for further information: Media: Ambre James-Brown +45 3079 9289abmo@ Liz Skrbkova (US)+1 609 917 0632lzsk@ Investors: Jacob Martin Wiborg Rode+45 3075 5956jrde@ David Heiberg Landsted +45 3077 6915 dhel@ Sina Meyer +45 3079 6656 azey@ Ida Schaap Melvold +45 3077 5649 idmg@ Frederik Taylor Pitter +1 609 613 0568fptr@ References1. Linglart A, Bottcher V, Rassmussen MH, et al. Weekly Somapacitan is Effective and Well-Tolerated in Children Born Small for Gestational Age: Randomised Phase 3 Trial. Oral presentation presented at the Joint Congress of ESPE and ESE 2025; 10-13 May 2025; Bella Center, Copenhagen, Denmark. JOINT4131.2. Jorge A, Albanese A, Rasmussen MH, et al. Weekly Somapacitan is Effective and Well-Tolerated in Children with Noonan Syndrome: Randomised Phase 3 Trial. Poster presentation presented at the Joint Congress of ESPE and ESE 2025; 10-13 May 2025; Bella Center, Copenhagen, Denmark. JOINT4118.3. Abuzzahab J, Dauber A, Rasmussen MH, et al. Weekly Somapacitan is Effective and Well-Tolerated in Children with Idiopathic Short Stature: Randomised Phase 3 Trial. Oral presentation presented at the Joint Congress of ESPE and ESE 2025; 10-13 May 2025; Bella Center, Copenhagen, Denmark. JOINT4048.4. Loftus J, Miller BS, Parzynski CS, et al. Association of Daily Growth Hormone Injection Adherence and Height Among Children With Growth Hormone Deficiency. Endocr Pract. 2022;28:565-571.5. Brod M, Hojbjerre L, Alolga SL, et al. Understanding Treatment Burden for Children Treated for Growth Hormone Deficiency. Patient. 2017;10:653-666.6. NCT05330325. A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow (REAL 8). Available at: Last accessed: May 2025.7. Definitive Healthcare. Basket trial. Available at: Last accessed: April 2025.8. NCT05723835. A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9 (REAL 9). Available at: Last accessed: May 2025.9. Sogroya® (somapacitan): SmPC. 2025 [online] Available at: Last accessed: May 2025.10. Sogroya® (somapacitan-beco injection) US PI. 2025 [online] Available at: Last accessed: May 2025.11. Johansson E, Nielsen AD, Demuth H, et al. Identification of Binding Sites on Human Serum Albumin for Somapacitan, a Long-Acting Growth Hormone Derivative. Biochemistry. 2020;59:1410-1419. Attachment PR250512-ESPE-ESE-2025-REAL8
Yahoo
12-05-2025
- Health
- Yahoo
Novo Nordisk A/S: Once-weekly Sogroya® (somapacitan) is an efficacious and well-tolerated long-acting growth hormone in children with growth disorders: results from REAL8 phase 3 basket study presented at the joint Congress of ESPE and ESE
The REAL8 trial showed that after 52 weeks, once-weekly Sogroya® (somapacitan) had similar clinical outcomes and safety profile to once-daily Norditropin® (somatropin) in children born small for gestational age (SGA)1, or with Noonan syndrome (NS)2, or with idiopathic short stature3. Superiority was achieved for once-weekly somapacitan versus daily growth hormone in children with NS2, as well as compared to lower doses of daily growth hormone in children born SGA1. These conditions are often associated with significant health challenges and a high treatment burden from daily injections4, which can lead to a lack of adherence and put successful treatment outcomes at risk. Novo Nordisk is committed to bringing our expertise and scientific innovation to help improve the lives of children with conditions that impact their growth. Bagsværd, Denmark, 12 May 2025 – Novo Nordisk today presented data from the phase 3 REAL8 basket study, which showed that once-weekly Sogroya® (somapacitan) was non-inferior to the once-daily growth hormone Norditropin® (somatropin) in improving yearly growth rate (as measured by height velocity [HV] at Week 52) in pre-pubertal children born small for gestational age (SGA)1, or with Noonan syndrome (NS)2, or with idiopathic short stature (ISS)3. In addition, superiority was achieved for once-weekly Sogroya® versus daily growth hormone in children with NS2, as well as compared to lower doses of daily growth hormone in children born SGA1. REAL8 data showed that Sogroya® was well-tolerated, with no safety or tolerability issues identified compared to once daily growth hormone1-3. Insulin-like growth factor 1 (IGF-1) response in patients treated with once-weekly Sogroya® was similar to those treated with daily growth hormone1-3. Results from the Turner syndrome (TS) sub-study of REAL8 will be available later this year. These data were presented as three late-breaking abstracts at the first Joint Congress of the European Society for Paediatric Endocrinology (ESPE) and European Society of Endocrinology (ESE) in Copenhagen, Denmark1-3. 'Children with growth failure face many health challenges beyond just being shorter than their peers. They often have metabolic disruptions and developmental difficulties that can seriously affect their wellbeing and quality of life, as well as long-term effects such as increased risk of cardiovascular disease or type 2 diabetes,' said Professor Agnès Linglart, Professor of Paediatrics at the Bicêtre Paris-Saclay University and Hospital, France, and one of the lead investigators on REAL8. 'The REAL8 data presented today marks an important step forward in providing these patients with an effective, once-weekly option that can potentially reduce treatment burden and improve adherence and treatment outcomes.' The REAL8 trial achieved its primary endpoints for the first three sub-studies, demonstrating that once-weekly Sogroya® was non-inferior to once-daily growth hormone treatment at Week 52 across the three indications presented: In children born SGA, Sogroya® demonstrated superior estimated mean HV when compared with a lower dose (0.035 mg/kg/day) of somatropin (11.0 vs 9.4 cm/year), and non-inferior estimated mean HV when compared with a higher dose (0.067 mg/kg/day) of somatropin (11.0 vs 11.1 cm/year)1. In children with NS, Sogroya® demonstrated superior estimated mean HV compared with somatropin (10.4 vs 9.2 cm/year)2. In children with ISS, Sogroya® demonstrated non-inferior estimated mean HV compared with daily somatropin (10.5 vs 10.5 cm/year)3. Non-adherence to growth hormone treatment is a common problem which puts successful treatment outcomes at risk. Daily injections can be a burden for children and their caregivers, leading to lack of adherence due to discomfort or pain at injection sites, inconvenience and disruption to daily life5. One study showed that missed daily injections resulted in a difference of 6.1 cm in height over 3 years when comparing nonadherent with adherent patients4. 'Treatment adherence is an issue when it comes to improving outcomes in children with growth failure. Imagine if a child misses only one day of treatment each week, amounting to 52 missed days per year. Over a seven-year treatment window, this results in one year of missed treatment and can have a significant knock-on impact on their health,' said Martin Lange, executive vice president for Development at Novo Nordisk. 'We are committed to providing a portfolio of growth hormone therapies with flexibility in administration timing and missed doses, which may better suit the needs of children with growth failure. These encouraging results from REAL8 mark a significant step forward in achieving that commitment.' In April 2025, based on the data from REAL8 and REAL9, the three indications (SGA, NS and ISS) were submitted for regulatory review in both the EU and US. About REAL8The REAL8 study is part of the ongoing REAL clinical trial programme, it is a randomised, open-label, active-controlled, parallel-group, phase 3 trial evaluating the efficacy and safety of once-weekly Sogroya® (somapacitan) in children born SGA, or with TS, NS or ISS. The primary treatment period was 52 weeks followed by a two-year safety extension phase6. REAL8 has an innovative basket trial design that is investigating once-weekly Sogroya® (somapacitan) in four different but related indications (SGA, TS, NS and ISS) under one trial protocol6. This trial design allows for a more efficient clinical development process by speeding up recruitment and consolidating resources7, potentially bringing treatment to patients with these conditions sooner. This is the first time a trial design of this type has been implemented in the growth disorder space. In REAL8, pre-pubertal children with NS, TS or ISS were randomised to receive either once-weekly Sogroya® (somapacitan) 0.24 mg/kg/week or once-daily somatropin 0.050 mg/kg/day; children born SGA were randomised to receive either somapacitan 0.24 mg/kg/week, or low dose of somatropin 0.035 mg/kg/day, or high dose of somatropin 0.067 mg/kg/day6. About REAL9The REAL9 study is part of the ongoing REAL clinical trial programme, it is a single-group assignment, phase 3 study evaluating the safety and efficacy of once-weekly Sogroya® (somapacitan) in children 10 years or older born SGA, or with TS, NS or ISS. The study will last for approximately 3 years8. About once-weekly Sogroya® Once-weekly Sogroya® (somapacitan) is a prescription human growth hormone analogue, similar to current daily growth hormone. It is currently approved for once-weekly treatment of children and adults who do not produce enough growth hormone9,10. Using albumin-binding prolongation technology, Sogroya® attaches to albumin, a protein in the blood, to help delay its removal from the body. This well-established technology has been used for over 20 years in Novo Nordisk's diabetes treatment. It allows for the growth hormone to work longer11. About Novo NordiskNovo Nordisk is a leading global healthcare company founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 77,400 people in 80 countries and markets its products in around 170 countries. For more information, visit Facebook, Instagram, X, LinkedIn and YouTube. Contacts for further information: Media: Ambre James-Brown +45 3079 9289abmo@ Liz Skrbkova (US)+1 609 917 0632lzsk@ Investors: Jacob Martin Wiborg Rode+45 3075 5956jrde@ David Heiberg Landsted +45 3077 6915 dhel@ Sina Meyer +45 3079 6656 azey@ Ida Schaap Melvold +45 3077 5649 idmg@ Frederik Taylor Pitter +1 609 613 0568fptr@ References1. Linglart A, Bottcher V, Rassmussen MH, et al. Weekly Somapacitan is Effective and Well-Tolerated in Children Born Small for Gestational Age: Randomised Phase 3 Trial. Oral presentation presented at the Joint Congress of ESPE and ESE 2025; 10-13 May 2025; Bella Center, Copenhagen, Denmark. JOINT4131.2. Jorge A, Albanese A, Rasmussen MH, et al. Weekly Somapacitan is Effective and Well-Tolerated in Children with Noonan Syndrome: Randomised Phase 3 Trial. Poster presentation presented at the Joint Congress of ESPE and ESE 2025; 10-13 May 2025; Bella Center, Copenhagen, Denmark. JOINT4118.3. Abuzzahab J, Dauber A, Rasmussen MH, et al. Weekly Somapacitan is Effective and Well-Tolerated in Children with Idiopathic Short Stature: Randomised Phase 3 Trial. Oral presentation presented at the Joint Congress of ESPE and ESE 2025; 10-13 May 2025; Bella Center, Copenhagen, Denmark. JOINT4048.4. Loftus J, Miller BS, Parzynski CS, et al. Association of Daily Growth Hormone Injection Adherence and Height Among Children With Growth Hormone Deficiency. Endocr Pract. 2022;28:565-571.5. Brod M, Hojbjerre L, Alolga SL, et al. Understanding Treatment Burden for Children Treated for Growth Hormone Deficiency. Patient. 2017;10:653-666.6. NCT05330325. A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow (REAL 8). Available at: Last accessed: May 2025.7. Definitive Healthcare. Basket trial. Available at: Last accessed: April 2025.8. NCT05723835. A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9 (REAL 9). Available at: Last accessed: May 2025.9. Sogroya® (somapacitan): SmPC. 2025 [online] Available at: Last accessed: May 2025.10. Sogroya® (somapacitan-beco injection) US PI. 2025 [online] Available at: Last accessed: May 2025.11. Johansson E, Nielsen AD, Demuth H, et al. Identification of Binding Sites on Human Serum Albumin for Somapacitan, a Long-Acting Growth Hormone Derivative. Biochemistry. 2020;59:1410-1419. 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Irish Independent
09-05-2025
- Health
- Irish Independent
Best friend makes urgent plea for Waterford mum with chronically ill baby boy
Baby Billy's severe condition has meant that he has not left hospital since being born in December 2023 and mum Rebecca Dalton has barely left either. Having been diagnosed with a rare genetic syndrome, baby Billy will need specialist care and an adapted home to leave hospital, while Rebecca is currently unable to leave his side to return to work, with bills mounting up. To support her best friend, Rachel Byrne has set up an online fundraising campaign, describing her friend's situation as 'heartbreaking'. 'She doesn't get out of the hospital much at all,' she added. 'Only very, very rarely. Since he's been in the hospital for 17 months now, she's actually only been out of the hospital twice overnight. And that was down to her home place.' Rachel told that she and Rebecca are old friends going back ten years or more to their days working in Waterford's Tower Hotel together. Rachel said they are still waiting to get home help for Rebecca and Billy, and more tests are pending. 'They are just waiting for results, until she knows more it's up in the air each week. There are always setbacks,' she said. Billy was born premature at 32 weeks on December 8, 2023. He was transferred to Crumlin after three days in Waterford University Hospital. Billy has been diagnosed with Noonan Syndrome, a rare genetic syndrome that affects many parts of the body. He has hypertrophic cardiomyopathy, pulmonary valve stenosis and a hole in his heart. Between December, 2023, and March, 2024, Billy suffered from cardiac arrest and respiratory arrest on three difference occasions, he was intubated numerous times, until the doctors came to the decision that he would need a tracheostomy. Billy was in ICU up until this point. Billy received his tracheostomy in April of 2024 and was transferred to the Transitional Care Unit. Multiple infections, including sepsis, viral respiratory infections and bacterial infections, have caused him to have many setbacks, resulting in him going up and down to ICU a further six times from May to November last year. In August of 2024 Billy underwent surgery for a PEG (percutaneous endoscopic gastrostomy) because of his feeding issues. Due to oral aversions and gut dysmotility this was a much needed surgery. 'But since, his gut issues have not subsided and it is what's keeping them in hospital at the moment,' said Rachel. 'They are looking at a further three plus months in hospital. 'Billy has not had the chance to have a normal start in life and has spent almost 17 months in hospital so far, and they are desperate to get home. 'Billy will need a full-time carer which means for the foreseeable Rebecca will not be able to return to work. She is Billy's sole carer.' Rachel said it's 'not looking great' on Thursday this week, as they are waiting on nurses at the moment to help Rebecca and Billy. 'She needs nurses to stay in the home in Waterford. That's why we've set up this GoFundMe. She was given a special house down in Waterford that will accommodate Billy when they do move home. 'She pays bills for that house now, and then she has to fully furnish it. Then she has to get all the machines that Billy is going to need. She will need proper equipment to set her up. 'She's also paying for the Ronald McDonald house that she's staying in every week as well. They supply food – they cook dinners, but she just stays in the hospital 24/7. She has to be there.' Still, Rebecca, is 'over the moon' that the campaign has raised over €2,100 in donations so far. "She was really struggling there,' she said. 'Her head was over the water, she really didn't know what she was going to do.' Rachel's fundraising campaign is available to donate at by searching for 'Rebecca and Billy'.
