Pompe disease is a rare genetic disorder resulting from GAA gene mutations, leading to acid alpha-glucosidase deficiency and glycogen buildup. Key symptoms include progressive muscle weakness and ...
LONDON, Oct. 01, 2025 (GLOBE NEWSWIRE) -- Spur Therapeutics today announced it will present new clinical data for avigbagene parvec (FLT201), its Phase 3-ready gene therapy candidate for Gaucher ...
Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.
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Hansa Biopharma’s Imlifidase Shows Promise in Gene Therapy for Crigler-Najjar Syndrome
Hansa Biopharma announced that Genethon will present preliminary data from a Phase 2 trial of imlifidase as a pre-treatment ...
Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The first test ...
Xanthine oxidase inhibitors, primarily allopurinol and febuxostat, are the mainstay of treatment. The goal of chronic gout management includes lowering serum urate levels to below 6 mg/dL. 5 Overall, ...
It’s a big day for Sanofi as the pharmaceutical giant welcomes two European drug approvals, both for rare enzyme deficiencies. One regulatory nod ushers in the first treatment in Europe for a ...
The enzyme replacement therapy market size is calculated at USD 11.34 billion in 2025 and is expected to reach around USD ...
The "Lysosomal Storage Disease Treatment Market Opportunity, Growth Drivers, Industry Trend Analysis, and Forecast 2025-2034" ...
In the United States about 5%-8% of the population is affected by an autoimmune disorder. Other reports, however, estimate a higher prevalence of various types of autoimmune disorders due to complex ...
Please provide your email address to receive an email when new articles are posted on . A phase 3 trial evaluating intrapleural enzyme therapy vs. video-assisted thoracoscopic surgery is feasible.
Changes in vision are often a common sign of aging. But what if we could reverse age-related visual decline? In a new study, ...
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