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For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
Researchers reduce cardiac arrhythmia after heart attacks using gene therapy
During a severe heart attack many heart muscle cells die and are replaced by scar tissue to stabilize the heart wall.
Sarepta's Shocking Stand: Company Rejects FDA Request To Pull Gene Therapy Elevidys
FDA places clinical hold on Sarepta's LGMD gene therapy trials after three deaths and revokes platform tech designation due to safety concerns.
News-Medical.Net on MSN1d
Breakthrough gene therapy reverses genetic deafness across age groups
Using gene therapy to treat hereditary deafness is safe and effective in both children and adults, according to new findings ...
Managed Healthcare Executive1d
Roctavian Gene Therapy Controls Bleeding for Five Years in Severe Hemophilia A
BioMarin's Roctavian gene therapy shows long-term effectiveness in treating severe hemophilia A, reducing bleeding and ...
CRISPR and Cancer: How Gene Editing Is Revolutionizing Oncology
Discover how CRISPR gene editing is creating personalized cancer therapies, enhancing immunotherapy, and offering new hope ...
MedPage Today on MSN3d
Gene Therapy May Hold Better Outcomes for Congenital Deafness Than Cochlear Implant
Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts ...
The Manila Times1d
Opus Genetics and the Global RDH12 Alliance Partner to Advance RDH12 Gene Therapy for Inherited Childhood Blindness
RDH12, a gene therapy for RDH12-associated Leber congenital amaurosis (RDH12-LCA) RESEARCH TRIANGLE PARK, N.C., July 23, 2025 ...