The management of achondroplasia, given its systemic nature, requires multidisciplinary cooperation to maximize outcomes. Achondroplasia is a genetic skeletal dysplasia that affects the development ...

The R&D head is looking for "clever science," which Friberg said is readily apparent in BioMarin’s recent research pact with ...

However, new investigational treatments are vying to compete in the area. Achondroplasia is a genetic bone disorder that causes short limbs and dwarfism. Although this growth disorder affects around ...

BioMarin is a global biotechnology company focused on developing and commercializing therapies for rare genetic diseases. BioMarin specializes in enzyme replacement therapies, gene therapies, and ...

Feb. 19, 2025 — Researchers have found a compound in ginger, called furanodienone (FDN), that selectively binds to and regulates a nuclear receptor involved in inflammatory bowel disease (IBD ...

The DWP Personal Independence Payments (PIP) can be claimed by those who suffer from a number of issues and you could get ...

Children with achondroplasia seem to be more challenged in the anterior-posterior direction, which might be linked to an anterior-shifted pelvis. No difference was observed in bipedal static postural ...

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

As of February 17, 2025, 1,028 unique patient prescriptions for Attruby™ have been written by 516 unique prescribers since FDA approval- ...