New partnership hopes to give BSS patients more options for treatment. Viralgen and Axovia Therapeutics are partnering to develop and manufacture a gene therapy for the treatment of retinal dystrophy ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

The atypical RNA forms clumps in muscle cells ... two types of myotonic dystrophy — the most common form of muscular dystrophy in which symptoms onset occurs during adulthood. Interim data from that ...

The methods and protocols generated in this study are accessible and can be implemented in any standard research laboratory without the need for ...

In this review of RNA therapies, we paint an overview of what’s going on in a field that is addressing a number of diseases.

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...

The data showed reduced difficulties in standing, walking and running that were statistically significant, the company said.

The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic ...

Roche has reported positive topline outcomes from the second year of the multinational Phase III EMBARK trial of Elevidys ...

New research highlights the promise of a novel stem cell treatment strategy for leptomeningeal brain metastasis (LBM), a ...

As the sports industry continues to evolve and push the boundaries of science and technology, athletes are achieving feats previously thought impossible—but t ...