Latest news with #achondroplasia
Yahoo
07-07-2025
- Business
- Yahoo
Ascendis to Share Its Latest Endocrinology Rare Disease Data at ENDO 2025
COPENHAGEN, Denmark, July 07, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced two oral presentations at ENDO 2025, the annual meeting of the Endocrine Society being held July 12-15, 2025, in San Francisco. Dr. Aliya Khan will present new data from Week 156 of the Company's Phase 3 PaTHway Trial demonstrating the safety and efficacy of long-term treatment with TransCon® PTH (palopegteriparatide) in adults with hypoparathyroidism – including maintenance of serum and urine biochemistries within normal levels and sustained improvement in renal function. Dr. Carlos Bacino will present additional safety and tolerability data from Week 52 of the Company's pivotal ApproaCH Trial of once-weekly TransCon® CNP (navepegritide) in children with achondroplasia. 'Ascendis is making important advances in its growing Endocrinology Rare Disease portfolio,' said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma. 'We are pleased to partner with renowned investigators during ENDO 2025 to share data demonstrating the long-term benefits and safety of our groundbreaking treatment for adults with hypoparathyroidism, as well as data that we believe supports the transformative potential of our investigational therapy for children with achondroplasia.' Hypoparathyroidism OR-10-05Saturday, July 122:15–2:30 PM PacificRoom 153 Oral PresentationEfficacy and Safety of Palopegteriparatide Treatment in Adults With Hypoparathyroidism: 3-Year Results from the Phase 3 PaTHway Trial Presented by Dr. Aliya KhanClinical Professor of Medicine, Division of Endocrinology & Geriatrics; Director of the Calcium Disorders Clinic at McMaster University Achondroplasia OR-36-05Monday, July 142:15–2:30 PM PacificRoom 308 Oral PresentationSafety and Tolerability of Navepegritide Treatment in Children With Achondroplasia: 52-Week Results from the Pivotal ApproaCH TrialPresented by Dr. Carlos BacinoProfessor, Molecular and Human Genetics at Baylor College of Medicine; Chief of Genetic Services at Texas Children's For more program information, please visit the ENDO 2025 website. About HypoparathyroidismHypoparathyroidism is an endocrine disease caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidney and indirectly on the intestine. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%), while other etiologies include autoimmune and idiopathic causes. About AchondroplasiaAchondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia vary across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, enlarged brain ventricles, impaired muscle strength and stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; many of these persist or worsen in adulthood. These medical complications can have detrimental effects on quality of life, physical functioning, and psychosocial function. Individuals with achondroplasia often require multiple surgeries and procedures to alleviate the condition's many complications. About Ascendis Pharma A/SAscendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit to learn more. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis' future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) the transformative potential of TransCon CNP for children with achondroplasia; (ii) Ascendis' ability to apply its TransCon technology platform to make a meaningful difference for patients; and (iii) Ascendis' application of its TransCon technologies to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis' products and product candidates; unforeseen safety or efficacy results in Ascendis' development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis' development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis' business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis' ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including tariffs and trade policies. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis' business in general, see Ascendis' Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 12, 2025, and Ascendis' other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, and TransCon are trademarks owned by the Ascendis Pharma group. © July 2025 Ascendis Pharma A/S. Investor Contacts: Media Contact: Sarada Weerasinghe Melinda Baker Ascendis Pharma Ascendis Pharma ir@ media@ Patti Bank ICR Healthcare +1 (415) 513-1284
Yahoo
13-06-2025
- Business
- Yahoo
TransCon® hGH Boosts TransCon® CNP Results in Kids with Achondroplasia by Week 26
Ascendis Pharma A/S (NASDAQ:ASND) is among the 13 Biotech Stocks with Huge Upside Potential. It reported interim Week 26 data from its Phase 2 COACH Trial, confirming that combining TransCon hGH (lonapegsomatropin) with TransCon CNP (navepegritide) raised annualized growth velocity (AGV) and height Z-scores in children with achondroplasia. A close-up view of a hand manipulating a syringe while delivering TransCon CNP into a tumor. A mean AGV of 9.14 cm/year and a +0.53 Z-score rise were attained by treatment-naïve youngsters (N=12). Children (N=9) treated with TransCon CNP in the past achieved an AGV of 8.25 cm/year with a +0.44 Z-score improvement. The majority of side effects were modest, and safety remained consistent with monotherapies. The COACH Trial is the first to assess this combination in kids between the ages of two and eleven. While TransCon CNP is undergoing FDA priority review, TransCon hGH is approved as SKYTROFA® for juvenile growth hormone insufficiency. The treatment focuses on the FGFR3-CNP signaling imbalance that is at the heart of achondroplasia, a disorder that affects more than 250,000 people worldwide. It is anticipated that the results of week 52 and a scheduled Phase 3 trial in Q4 2025 will further clarify efficacy and long-term benefits. The biopharmaceutical business Ascendis Pharma A/S (NASDAQ:ASND) uses its TransCon technology platform to significantly improve patient outcomes. While we acknowledge the potential of ASND as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 10 High-Growth EV Stocks to Invest In and 13 Best Car Stocks to Buy in 2025. Disclosure. None.
RNZ News
19-05-2025
- RNZ News
Man jailed in Tonga after throwing a child against a wall
Photo: 123rf A man has been jailed in Tonga after throwing a six-year-old child - who has a bone growth disorder - against a wall. Matangi Tonga reported the defendant pleaded guilty to common assault and causing serious bodily harm. Justice Tupou said the charges did not reflect what, by his own admission, the defendant did. "He punched and kicked the child until his mouth bled, strangled him by the throat until his eyes almost fell out and then threw him against the wall. Those in my view are acts of a depraved mind, especially when his victim was a six-year-old achondroplasia child that was no match for him," she said. "Meanwhile, the defendant - in an attempt to justify his actions - gave three different reasons to different people. He told [the child's mother] it was because her family were gossiping about them; he told the police he was angry with [the child's mother] and while he was trying to calm down he saw [the child's] soiled nappy leak; and lastly, he told the probation officer that he was mad and angry with [the child's mother] for playing with his feelings." The defendant submitted to court that he had conveyed his apology and regretted his actions; he took responsibility for his behaviour and said he was aware of the consequences, and he had made a vow not to be violent and is taking steps to ensure what happened is not repeated. But the judge was not convinced. "I find it unacceptable that he is more concerned about any disruptions to his studies, as a result of his offending, over the damage he may have caused to the vulnerable victim. For those reasons I doubt his remorse." The defendant was sentenced to two and a half years' imprisonment, with the final 12 months of the sentence suspended for two years with conditions. It is another chapter in an epidemic of violence in the Pacific. The United Nations warns that Pacific children face alarmingly high rates of violence and abuse , which are among the highest in the world. At the inaugural Pacific Child Wellbeing Summit earlier this year, UNICEF Pacific's representative Jonathan Veitch said boys and girls in the region face significant challenges that threaten their wellbeing. He also called for increased regional collaboration and resource-sharing to strengthen child protection systems. "It is not only the right thing to do but also the smart thing to do. The impact of child protection on a country's economic and social development is significant." The Samoa Victim Support Group (SVSG) said it has noted an increase in child victims being abused sexually and violently. "It is particularly disheartening to witness young girls, as young as 8 and 9 years old, being sheltered as victims of sexual abuse by their own relatives," it said in a statement earlier this month. "One such case involves a young girl currently under SVSG's care, who was discovered by a family sleeping in front of their home." According to SVSG, the girl had been a victim of abuse and felt too frightened to return to her abusive home environment. "The public was also devastated to hear about the death of a 13-year-old girl who was sexually abused and killed by her own relative at her own home." Unicef said up to 80 percent of children in Papua New Guinea experience physical or emotional violence and widespread neglect . Last month, alleged cases of domestic violence in Fiji led to the deaths of three people . In November 2024, a UN Women report stated the prevalence of reported physical and/or sexual intimate partner violence - taking place in the previous 12 months - was 30 percent in Melanesia.
Medscape
09-05-2025
- Health
- Medscape
Fast Five Quiz: Management of Achondroplasia
Achondroplasia is the most common form of skeletal dysplasia and is associated with numerous complications that require careful monitoring and management. This form is typically diagnosed at birth or during infancy; treatment can begin around this point to mitigate future complications, though achondroplasia usually requires lifelong management. Surgical interventions are often necessary to address specific complications, and recent advances in pharmacologic treatment have also been added to the management of this condition. Regardless, achondroplasia still impacts quality of life. Do you know how to effectively manage achondroplasia? Check your knowledge with this quick quiz.
