CRISPR-Cas9, originally discovered in prokaryotic organisms to be part of their immune system, has rapidly developed into a gene editing tool far more accurate than its predecessors. This technology is thus generating a lot of buzz due to its potential to correct genetic diseases. However, what if it could be used to identify factors that can lessen disease severity? These targets could be the source...

Expressing his hardships and triumphs while battling facioscapulohumeral muscular dystrophy, Bonita eighth-grader Sam Ray is one of the winners of the national Scholastic’s annual essay contest.The annual essay, “Breaking Barriers: In Sports, In Life,” was created by Scholastic and Major Baseball League in honor of...

Article originally appeared on sciencedaily.com, November 7, 2017. In a recent paper published in the journal Skeletal Muscle, a Saint Louis University researcher reports success in identifying new drug targets that potentially could slow or halt the progression of a form muscular dystrophy, an illness characterized by progressive muscle...

Article originally appeared on musculardystrophynews.com, October 10, 2017. Resolaris (ATYR1940) improved the muscle strength of nearly two-thirds of adolescents and young adults with early-onset facioscapulohumeral muscular dystrophy (FSHD), according to final results of a Phase 1/2 clinical trial. The therapy’s maker, aTyr Pharma, presented the findings at the 22nd International Annual Congress of the World Muscle Society...