Discover the current pipeline candidates advancing through the clinic for the treatment of sickle cell disease.

Sarepta Therapeutics Inc. is cutting more than one-third of its workforce and will add a black box warning label to its gene therapy for a fatal muscle disorder after two patient deaths raised doubts ...

The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated ...

An expert discusses how high-cost gene therapies require evidence standards including Phase 3 trial data showing complete response rates and durability, real-world evidence and health economic ...

An expert discusses how recent updates to AUA, EAU and NCCN guidelines have cautiously shifted toward more bladder-sparing approaches for BCG-unresponsive patients, incorporating new FDA-approved ...

The favorable market reaction indicates investor relief at management’s swift measures to rectify the company's worsening ...

It is trying to treat a devastating lifelong disease by fixing it at its source. We are able to fix the actual genetic ...

Sarepta Therapeutics said it will eliminate 36% of its workforce—approximately 500 jobs—in a restructuring that follows the ...

Sarepta Therapeutics shares surged 34% in after-hours trading on Wednesday after the company said it would slash 500 jobs, or ...

Boyang Wang on Targeting Underfunded Longevity Projects SINGAPORE, SG / ACCESS Newswire / July 16, 2025 / Immortal Dragons, a ...

Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...

Fragile X disorders include two distinct conditions: fragile X syndrome (the most common single-gene cause of developmental delay and autism) and an entirely separate group of premutation syndromes.