The company said that more than 800 people have been treated with Elevidys, with only one experiencing liver failure.

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the death Tuesday morning, sending the company's shares plummeting in morning ...

Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.

While plenty of people are going green in honor of Saint Patrick's Day, one student at East Stroudsburg University is turning ...

The Phase I/II Deliver trial sought to establish the impact of DYNE-251 on several mobility-related endpoints, seeing an ...

The Food and Drug Administration has accepted Capricor Therapeutics' application seeking approval of its cell therapy deramiocel for muscular dystrophy patients and granted it a priority review.

Aside from muscular dystrophy, other examples of rare diseases include hemophilia, an inherited blood disorder that causes excessive bleeding and bruising; phenylketonuria, an inherited disorder ...

Preventive treatment with standard heart medications — when given before the onset of cardiac troubles — extends survival among males with Duchenne muscular dystrophy (DMD), according to data from the ...

Jang Ik-sun, a 37-year-old student from South Korea, has achieved an extraordinary academic milestone despite living with muscular dystrophy, a condition that has left him almost entirely ...

Get Instant Summarized Text (Gist) Nanoparticles have been developed to deliver microRNAs to muscle stem cells, offering a potential treatment for Duchenne muscular dystrophy. These nanoparticles ...

This is the first human trial of gene supplementation therapy targeting this condition. Retinal dystrophy caused by biallelic variants in the AIPL1 gene leads to severe visual impairment from ...