Taysha Gene Therapies announced that it has joined with Catalent, a pharmaceutical services provider, to support production of its…
Articles by Forest Ray, PhD
The U.S. Food and Drug Administration (FDA)Â has given a favorable opinion of the continued preclinical development plan for BBDF-101,…
Cannabinoids were used to successfully treat seizures in a patient with Batten disease for whom conventional therapies alone…
The European Medicines Agency (EMA) has given priority medicines (PRIME) status to AT-GTX-501, Amicus Therapeutics‘ investigational gene therapy…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography…
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the…
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure…
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in…
Those afflicted with lysosomal storage disorders such as Batten disease may benefit from a clever new treatment strategy one day.