Latest news with #Aldurazyme
IOL News
30-05-2025
- Health
- IOL News
Alyssa's fight: Parents launch campaign for urgent treatment of rare condition
Somerset Park couple raises funds for daughter diagnosed with MPS I. Image: Supplied A young couple from Somerset Park, KwaZulu-Natal, has launched a desperate effort to secure life-saving treatment for their infant daughter, Alyssa Cas Aldworth, after she was diagnosed with a rare and potentially fatal genetic disorder. Alyssa, born on September 28, 2024, was recently diagnosed with Mucopolysaccharidosis Type 1 (MPS I), a rare condition caused by the absence of the IDUA gene. The gene is responsible for producing an enzyme that breaks down complex sugars in the body. Without treatment, children with MPS I rarely live past the age of ten. Her father, 29-year-old Trysten Aldworth, described the news as devastating. "Hearing that your only daughter has a rare genetic disorder with an extremely high mortality rate has broken us beyond words," he said. "It's something no parent should ever have to hear." Alyssa, affectionately known as Ally, spent her first 10 days in a neonatal intensive care unit due to severe heart and lung complications. Despite initial health setbacks, she has continued to show signs of improvement with regular medical monitoring. The condition requires immediate medical intervention in the form of Enzyme Replacement Therapy (ERT), using a drug called laronidase (Aldurazyme). However, the therapy comes at a steep cost, quoted at R78,064.64 per month, excluding VAT and must be administered weekly for life. Even with ERT, the enzyme cannot cross the blood-brain barrier, limiting its effectiveness in halting neurological damage caused by MPS I. As a result, doctors are urgently considering a Hematopoietic Stem Cell Transplantation (HSCT), a more aggressive treatment aimed at halting disease progression if performed within the first two years of life. The cost of HSCT abroad is estimated between £150,000 and £200,000 (approximately R3.6 million to R4.9 million), though the family hopes to explore a more affordable option within South Africa. A consultation with a haematologist is scheduled this week to assess the feasibility of the treatment locally. Video Player is loading. Play Video Play Unmute Current Time 0:00 / Duration -:- Loaded : 0% Stream Type LIVE Seek to live, currently behind live LIVE Remaining Time - 0:00 This is a modal window. Beginning of dialog window. Escape will cancel and close the window. Text Color White Black Red Green Blue Yellow Magenta Cyan Transparency Opaque Semi-Transparent Background Color Black White Red Green Blue Yellow Magenta Cyan Transparency Opaque Semi-Transparent Transparent Window Color Black White Red Green Blue Yellow Magenta Cyan Transparency Transparent Semi-Transparent Opaque Font Size 50% 75% 100% 125% 150% 175% 200% 300% 400% Text Edge Style None Raised Depressed Uniform Dropshadow Font Family Proportional Sans-Serif Monospace Sans-Serif Proportional Serif Monospace Serif Casual Script Small Caps Reset restore all settings to the default values Done Close Modal Dialog End of dialog window. Advertisement Next Stay Close ✕ Ad loading The couple launched a campaign on Back a Buddy with the aim to raise R468,000 and have raised close to R206,000. Aldworth who has been married to his wife Jade for five years, said their community's support has been overwhelming. "In just days, the amount of people who have called, messaged, donated or shared Ally's story has been nothing short of amazing. It's scary, but in the best possible way." Ally's middle name, Cas, honours Jade's late father, Craig Andrew Suttie, who died in July 2023 from cerebral malaria at the age of 47. The family, together since 2013, remains hopeful as they navigate complex medical decisions. "We just want what every parent wants; for our child to be healthy, to live a full life, and one day walk down the aisle," Trysten added. "We're going to do everything in our power to make that possible." IOL News Get your news on the go, click here to join the IOL News WhatsApp channel.
Yahoo
20-05-2025
- Business
- Yahoo
BioMarin Offers to Buy Inozyme for $270M to Boost Enzyme Therapy Biz
BioMarin Pharmaceutical BMRN announced that it has entered into a definitive agreement to acquire all outstanding shares of the clinical-stage company Inozyme Pharma INZY for $4.00 per share in cash, totaling nearly $270 million. The deal, approved by the board of directors of both companies, is expected to be closed in the third quarter of 2025. Following the completion of this acquisition, BioMarin will add Inozyme's lead asset, INZ-701, to its pipeline. This investigational enzyme replacement therapy (ERT) is currently being evaluated in a pivotal late-stage study for the treatment of a rare genetic disorder, ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency, in children. This condition affects blood vessels, soft tissues and bones, and is associated with increased cardiovascular mortality risk. Interim results from this late-stage study are expected in early 2026. If this data is positive, a regulatory approval for INZ-701 is expected in 2027, making it the first-in-disease treatment for this indication. Inozyme is also evaluating the ERT in a pivotal study for infants with ENPP1 and plans to initiate a supportive study for adolescents and adults with the disease. Inozyme is also exploring the potential of INZ-701 in separate clinical studies for two more rare disorders, namely ABCC6 Deficiency and calciphylaxis. Like ENPP1, there are currently no approved therapies for either of these disorders. Following this news on Friday, shares of Inozyme surged 178% and those of BioMarin gained about 2%. Year to date, shares of BMRN have lost 10%, while those of INZY have risen 43%. During the same timeframe, the industry has declined 7%. Image Source: Zacks Investment Research In our opinion, the deal is a strategic fit for BioMarin, which already markets five first-in-disease enzyme therapies, namely Aldurazyme, Brineura, Naglazyme, Palynziq and Vimizim. The addition of Inozyme's lead candidate will expand BioMarin's Enzyme Therapies portfolio and further diversify its revenue stream. The deal also benefits INZY as it lacks the commercial infrastructure and expertise needed to bring a drug to market, areas where BioMarin is already well established. BioMarin has reaffirmed its full-year 2025 guidance of $3.1-$3.2 billion for sales and $4.20-$4.40 for adjusted EPS. BioMarin currently carries a Zacks Rank #3 (Hold). BioMarin Pharmaceutical Inc. price | BioMarin Pharmaceutical Inc. Quote A couple of better-ranked stocks from the industry are Adaptive Biotechnologies ADPT and Agenus AGEN, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. In the past 60 days, estimates for Adaptive Biotechnologies' 2025 loss per share have improved from 92 to 87 cents. During the same timeframe, estimates for 2026 loss per share have narrowed from 69 to 65 cents. Adaptive Biotechnologies' earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 21.38%. Shares of ADPT have surged 51% year to date. Estimates for Agenus' 2025 loss per share have narrowed from $7.05 to $2.78 over the past 60 days, and the same for 2026 loss has improved from $7.14 to $5.26. Agenus' earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 22.71%. Year to date, its shares have gained 27%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report Agenus Inc. (AGEN) : Free Stock Analysis Report Adaptive Biotechnologies Corporation (ADPT) : Free Stock Analysis Report Inozyme Pharma, Inc. (INZY) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
