achondroplasia

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emerging-therapies-for-achondroplasia-treatment
Achondroplasia Treatment Space: Hunt for Potential Curative Therapies

Achondroplasia is a rare genetic disorder affecting bone growth, causing significant short stature, known as dwarfism. This condition is attributed to a fibroblast growth factor receptor 3 (FGFR3) gene mutation. The mutation results in an overactive FGFR3 gene, leading to a slowdown in bone formation within the car...

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Pharma News for Pfizer, UCB, BioMarin
FDA Approves PENBRAYA for Most Common Serogroups Causing Meningococcal Disease; BIMZELX Approved Moderate to Severe Plaque Psoriasis; FDA Approves BioMarin’s VOXZOGO; FDA Fast Track Designation to ANPD001 for Parkinson’s Disease; UCB Announces FDA Approval of ZILBRYSQ; EMA Granted Orphan Drug Designation to Lisata’s LSTA1

UCB announces FDA approval of ZILBRYSQ for the Treatment of Adults with Generalized Myasthenia Gravis On the 17th of October 2023, UCB (Euronext Brussels: UCB) made an announcement regarding the approval of ZILBRYSQ® (zilucoplan) by the US FDA for the management of generalized myasthenia gravis (gMG) in adult pa...

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