GlobalData on MSN
FDA puts REGENXBIO gene therapy trials on hold after brain tumour
A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.
The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
MedPage Today on MSN
FDA Halts Two Gene Therapy Trials After Child Develops Brain Tumor
Studies of Hunter and Hurler syndromes on hold ...
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...
Stocktwits on MSN
Rare stock slips 3% today — what’s up with Ultragenyx’s gene therapy for Sanfilippo syndrome?
Ultragenyx now expects a 6-month review period for the therapy and a likely decision from the FDA regarding potential ...
Stocktwits on MSN
RGNX shares pare losses as Wall Street dismisses gene therapy clinical hold spurred selloff as an overreaction
Leerink said the premarket selloff in shares underscores the degree of surprise on the news, feeding into the "FDA ...
By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...
Future GLP-1 gene therapy could enable long-term hormone production, with Fractyl Health and others planning human trials in 2024.
Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...
ACM impedes the heart from pumping blood to the rest of the body, and is a leading cause of sudden cardiac death in young ...
GlobalData on MSN
Genespire to take rare disease gene therapy to clinic in 2026
GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.
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