rare disease
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.
In Brief This Week: 23andMe, OS Therapies, Zai Lab, Genetic Alliance, Galatea Bio, Mursla Bio
News items for the week of April 21, 2025.
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.
The Sunshine Genetics Act would establish a consortium that plans to sequence 10,000 newborns for up to 600 rare conditions.
Apr 11, 2025
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Mar 26, 2025