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genetic disorder

Showing 3 posts of 3 posts found.

Hansa Biopharma announces positive data for Duchenne muscular dystrophy gene therapy

August 4, 2025
Research and Development Duchenne Muscular Dystophy, Hansa Biopharma, Rare Diseases, Sarepta Therapeutics, clinical trial, genetic disorder

Hansa Biopharma has announced encouraging results from its ongoing SRP-9001-104 trial, which is investigating the use of imlifidase as a …

News
sobi

Sobi says US FDA approves higher dose of Orfadin to treat rare genetic disorder

June 15, 2016
Research and Development, Sales and Marketing US FDA, genetic disorder, regulation, sobi

Swedish Orphan Biovitrum AB (Sobi) on Wednesday said the US Food and Drug Administration (FDA) has approved a higher strength …

News

Amicus Therapeutics’ Fabry disease drug gets European regulatory support

April 4, 2016
Research and Development, Sales and Marketing Amicus Therapeutics, EMA, Fabry disease, genetic disorder, rare disease

Shares in biotechnology firm Amicus Therapeutics (Nasdaq: FOLD) rose to close up nearly 3% after the European regulators announced backing …

News
The Gateway to Local Adoption Series

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STADA and Bio-Thera’s Gotenfia receives positive CHMP opinion for autoimmune diseases

December 18, 2025

Norgine enters exclusive licensing agreement with Vir Biotechnology

December 18, 2025

Incyclix appoints Kimberly Blackwell to Board of Directors

December 17, 2025

Hansa Biopharma appoints Max Sakajja as Vice President, General Manager Europe and International

December 17, 2025

GSK’s Exdensur receives MHRA approval for asthma and rhinosinusitis

December 16, 2025