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 ...

FSHD is the second most common form of muscular dystrophy after the Duchenne type, and affects around 1 million people worldwide, with no approved treatments ... large B-cell lymphoma (DLBCL).

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.

Ottawa scientist and palliative care physician Dr. Paul Hébert has served in many roles during his career: clinical scientist ...

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

has granted Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1). DYNE-101 is currently being evaluated in the ongoing Phase 1/2 ACHIEVE global clinical trial.

(RTTNews) - Dyne Therapeutics, Inc. (DYN), Tuesday announced that the U.S. Food and Drug Administration has granted Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type ...

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