Del-desiran reduced DMPK mRNA levels, but led to two serious adverse events ...

Cellular models used to search for new therapies for myotonic dystrophy type 1 do not usually take into account the diversity of subtypes presented by patients. A study led by researchers from the ...

Lamotrigine, a drug commonly used to treat epilepsy and certain mood disorders, has been shown to be an excellent treatment option for a rare genetic neuromuscular disease known as non-dystrophic ...

Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that ...

Cellular models used to search for new therapies for myotonic dystrophy type 1 do not usually take into account the diversity of subtypes presented by patients. A study led by researchers from the ...

Avidity accelerates global Phase 3 HARBOR™ study initiation to Q2 2024 following regulatory agreement on study design; primary endpoint is video hand opening time (vHOT) and secondary endpoints ...

Thurman M. Wheeler, MD, of the Department of Neurology at Massachusetts General Hospital, is the senior author of a paper published in Nature Communications, "Analysis of human urinary extracellular ...

WALTHAM, Mass.--(BUSINESS WIRE)--Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced that ...

Delpacibart etedesiran data from MARINA-OLE™ showed reversal of disease progression in multiple functional measures in DM1 compared to END-DM1 natural history data "We are pleased that the FDA has ...

Myotonic dystrophy type 1 (DM1) is a rare, progressive genetic disease that causes severe muscle weakness and other debilitating symptoms, such as compromised respiration and cardiac conduction ...

A recent discovery of a molecular connection between autism and myotonic dystrophy, a type of neuromuscular disease, may provide a breakthrough on how clinicians approach autism spectrum disorder. The ...