genetic disorder
Hansa Biopharma announces positive data for Duchenne muscular dystrophy gene therapy
August 4, 2025Research 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 …
Sobi says US FDA approves higher dose of Orfadin to treat rare genetic disorder
June 15, 2016Research 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 …
Amicus Therapeutics’ Fabry disease drug gets European regulatory support
April 4, 2016Research 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 …
