Inherited Disease
News on Mendelian diseases, hereditary cancer, variant classification, cystic fibrosis testing and more.
FDA Approves Abeona's Zevaskyn, EC Authorizes Krystal's Vyjuvek for Rare Skin Disease
Both gene therapies were approved as treatments for dystrophic epidermolysis bullosa, a rare and debilitating genetic skin condition.
BlackfinBio Launching Gene Therapy Trial in Rare Neurological Condition at Boston Children's
The UK-based company will evaluate BFB-101 in up to five children with hereditary spastic paraplegia type 47 in a Phase I/II trial.
Alcyone Therapeutics Gets Funding, Batten Disease Gene Therapy From Nationwide Children's Hospital
The biotech and hospital have expanded their collaboration, with Alcyone now advancing a gene therapy for Batten disease that the hospital was developing.
NICE Recommends Pharming Group's Joenja for Activated Phosphoinositide 3-Kinase Delta Syndrome
Joenja will be the first drug specifically for APDS to be reimbursed within the NHS and is available immediately in England and soon will be in Wales.
FD Foundation Spinout Tikun Therapeutics Charges Ahead Studying Therapies for Familial Dysautonomia
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The New York-based public benefit corporation is advancing three drug candidates for the extremely rare condition.