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...
https://youtu.be/LXr1L4NOmos ...
Justin Cohen is a remarkable young researcher, living with FSHD, and efforting to find a cure for the disease. The Carrino Foundation is proud to make his work possible. Here is Justin’s story, in his own words. ...
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...
There are approximately 15 steps that Chris Carrino will need to navigate in order to reach his broadcast position inside Wells Fargo Center. It may not seem all that daunting, but a small flight of stairs is physically challenging and potentially dangerous for someone living...
Be sure to mark your calendars now! Our ninth annual dinner dance will take place on Thursday, July 18, 2019, at Russo's on the Bay in Howard Beach, NY. Details and ticket information will be available soon. Hope to see you there!...
Facio Therapies announced today that it has selected its first series of potential FSHD drug development candidates. This series comprises several families of small-molecule compounds that are associated with a narrowly defined target class. Neither the compound families nor the target class have ever been...
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...
Bruce Beck of NBC 4 provides coverage of the C2C4FSHD bike tour....