The FDA has granted orphan drug designation to Cure Rare Disease’s CRD-002, an antisense oligonucleotide therapeutic for the treatment of spinocerebellar ataxia (SCA), including spinocerebellar ataxia ...
News Medical on MSN
California Grants $7.4 Million to Advance Gene-Edited Stem Cell Therapy for Friedreich’s Ataxia
Funding supports safety studies, manufacturing and clinical planning needed before applying to test the treatment in patients ...
Tiffany Fransen, diagnosed with Friedreich's ataxia, faces challenges from this rare disease. The genetic disorder affects ...
MINNEAPOLIS, Sept. 25, 2025 /PRNewswire/ -- The National Ataxia Foundation (NAF) today announced the launch of The Science Guy: Back in the Lab for FA, a new educational campaign to raise awareness ...
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