资讯

STAT1 小时
Patient dies in Sarepta gene therapy trial, adding to safety concerns
A 51-year-old man died last month after receiving an experimental treatment from Sarepta, the third death this year tied to ...
MedPage Today on MSN1 天
Duchenne Gene Therapy Will Undergo Changes After Patient Deaths
At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...
BioSpace1 天
Sarepta Tags DMD Gene Therapy Elevidys With Black Box Warning, Axes 500 Staff
Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...
Muscular Dystrophy News20 小时
Dosing begins in gene therapy trial for DMD-linked heart disease
The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated ...
Fierce Pharma3 天
Ultragenyx takes another hit as FDA snubs AAV gene therapy over manufacturing issues
The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...
Sarepta Says Another Patient Died After Receiving Gene Therapy
Sarepta Therapeutics Inc. said another patient has died from acute liver failure after receiving one of its gene therapies, putting additional pressure on the biotech company after the recent deaths ...
FDA rejects Ultragenyx gene therapy over manufacturing concerns
While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...
BioPharma Dive1 天
Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks
On a conference call, CEO Doug Ingram said the decision to cut 36% of the company's staff and halt several drug programs was ...
Managed Healthcare Executive1 天
CMS Launches Value-Based Gene Therapy Program for Sickle Cell Disease
CMS is brokering outcomes-based agreements on behalf of Medicaid programs. Thirty-three states, along with Washington, D.C., ...
Fierce Healthcare2 天
CMS officially names participants in Cell and Gene Therapy Access Model
The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...
The Manila Times2 天
ZEVASKYN™ Gene Therapy Now Available at New Qualified Treatment Center in San Francisco ...
- ZEVASKYN is the only FDA-approved therapy to treat RDEB wounds with a single application - ...
Cystic Fibrosis Foundation1 天
Cystic Fibrosis Foundation Commits Up to an Additional $24 Million for Prime Medicine to ...
Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million in Prime Medicine to continue ...