The Chris Carrino Foundation, in conjunction with Friends of FSHD Research, is proud to continue funding for the Dr Suja Janaggathan Lab at The University of Colorado. We have just approved funding of year two of this project, with our portion amounting to $46,344.50.
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 of novel treatments. A recent study out in Science Translational Medicine led by Angela Lek PhD and Louis Kunkel PhD explored this in Facioscapulohumeral Muscular Dystrophy (FSHD), a type of muscular dystrophy for which there is no treatment or cure.
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 Jackie Robinson, to inspire students by Robinson’s perseverance and to teach them how to use the same values he did to overcome hardships within their lives.
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 linked to FSHD in the scientific literature.
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 degeneration. Francis M. Sverdrup, Ph.D., research fellow in the …
Read more “FSHD NEWS: New drug targets for a form of muscular dystrophy”
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 (WMS) in Saint Malo, France, according to a press release. The …
Post by Chris Carrino June 20th is World FSHD Day. A day of awareness for FSHD, to show we are united for a cure. However, for those of us affected by this disease, June 20th is simply known as – Tuesday. There isn’t a moment of every day that I’m not acutely aware …
This story was originally published on musculardystrophynews.com on April 27, 2017. Resolaris, a protein therapy developed for rare muscle diseases by aTyr Pharma, improved muscle strength and quality of life in a small study of patients with early onset facioscapulohumeral muscular dystrophy (FSHD). These findings, as well as others regarding the effect of Resolaris in patients with …
Apabetalone (RVX-208) may become a promising therapy to treat facioscapulohumeral muscular dystrophy (FSHD), according to results announced by the pharmaceutical company Resverlogix. Researchers found this drug acts by switching off the DUX4 gene, which is abnormally active in the muscles of FSHD patients, causing inflammation and muscle atrophy. Apabetalone belongs to a group of …
Read more “FSHD NEWS: Apabetalone May Repress Toxic Gene in Rare Muscular Dystrophy (FSHD)”
Starting at the Space Needle in Seattle, our group of cyclists will steadily make their way across the country, covering a grand total of 4,200 miles until they cross the Brooklyn Bridge just 54 days later. The riders will average about 92 miles per day while traversing 15 states and visiting 46 cities along the way.