STAT
Child dies after receiving new experimental gene therapy
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
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Child dies in Phase I Capsida gene therapy trial
Capsida Biotherapeutics has paused an early-stage rare disease trial evaluating its lead gene therapy candidate, CAP-002, after the death of the first patient dosed in the study. The death comes just ...
FDA tightens Duchenne gene therapy after deaths of two teens
The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died.
FiercePharma
FDA restricts Sarepta's gene therapy Elevidys after Duchenne patient deaths
The FDA has officially limited the label of Sarepta Therapeutics’ Elevidys, putting an end to a whirlwind few months that saw the abrupt departure—and reinstatement—of top agency official Vinay Prasad ...
Heartland
FDA Halts, Then Resumes Gene Therapy After Three Deaths
After the deaths of three patients, the maker of an investigational gene therapy stopped shipments of the drug after it refused a request from the U.S. Food and Drug Administration (FDA) to pull the ...
Medpage Today on MSN
Gene Therapy Gets FDA's Strongest Warning After Fatal Liver Injuries
The FDA added a boxed warning to delandistrogene moxeparvovec (Elevidys) gene therapy for Duchenne muscular dystrophy and changed the indication of the drug after reports of fatal liver injury emerged ...
Fierce Pharma
Sarepta gets FDA's go-ahead to study immunosuppressive Elevidys regimen in bid to reverse label restriction
When the FDA reworked the prescribing information for Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) gene therapy Elevidys earlier this month, the company touted a plan to study a regimen | ...
The American Journal of Managed Care
FDA Approves Gene Therapy Onasemnogene Abeparvovec for Broader SMA Population
Onasemnogene abeparvovec-brve is the first gene replacement therapy approved for SMA patients aged 2 years and older, expanding access beyond infants. Phase 3 trials demonstrated significant motor ...
Benzinga.com
Child dies after receiving new experimental gene therapy
The first child to receive a new experimental gene therapy for a rare neurological disorder died a few days after dosing, the company sponsoring that trial said Wednesday. The company, Capsida ...
FierceBiotech
Capsida pauses phase 1 gene therapy trial after child dies
Capsida Biotherapeutics has paused a gene therapy trial after the first patient died. The biotech, which began the phase 1/2 study in July, took the action to give it time to determine the “root cause ...
Benzinga.com
STAT+: Child dies after receiving new experimental gene therapy
The first child to receive a new experimental gene therapy for a rare neurological disorder died a few days after dosing, the company sponsoring that trial said Wednesday. The company, Capsida ...