4D Molecular Therapeutics (4DMT) has said a trial of its gene therapy for Fabry disease has been placed on a clinical hold by the FDA, a few weeks after it reported three
Ipsen has suffered a setback in its marathon effort to bring palovarotene to market for ultra rare disease fibrodysplasia ossificans progressiva (FOP), after EU advisors recommended against
Alnylam's gene-silencing drug Amvuttra has been recommended as a treatment for hereditary transthyretin-related (ATTR) amyloidosis by NICE, paving the way for the drug to be made available
New drugs for immunological and inflammatory diseases feature prominently among new product launches this year that could top $1 billion in annual sales by 2027 or be clinical "game changer
Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe
In a new white paper from the World Without Disease initiative, a 2024 update is provided of the current endeavours and developments that have occurred since inaugural discussions last year