The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
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 ...
MedPage Today on MSN
FDA Halts Two Gene Therapy Trials After Child Develops Brain Tumor
Studies of Hunter and Hurler syndromes on hold ...
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.
University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...
A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...
By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...
The EMA’s positive opinion on Kygevvi marks a turning point for patients with TK2d, an ultra-rare genetic muscle disease previously managed only with supportive care.
Pharmaceutical Technology on MSN
Sensorion announces $71.9m reserved offering to expand gene therapy pipeline
Sanofi’s participation will support the progression of Sensorion’s genetic medicine pipeline.
After finding an intraventricular tumor in one clinical trial participant, the FDA placed a clinical hold on an experimental ...
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