Volunteers needed for Univ. of Minnesota studies

STUDY #1 Your participation is essential to advancing research on FSHD. Participants are sought for a University of Minnesota study on muscle stem cells in FSHD. The principal investigators are Michael Kyba, PhD, and Peter Karachunski, MD. What is involved? Study participants (FSHD and control individuals) will choose to provide any one or all of the following … Read more of “Volunteers needed for Univ. of Minnesota studies” »

Fulcrum Therapeutics raises $80 million in series B

Here’s some encouraging news about Fulcrum Therapeutics. The Cambridge, Massachusetts-based biotech that is developing a drug to treat Facioscapulohumeral muscular dystrophy has announced it has closed $80 million in series B financing. The company hopes to go public early next year, after filing papers with the FDA to begin human testing of its first drug, … Read more of “Fulcrum Therapeutics raises $80 million in series B” »

FSH Society launches national chapter program

Today, The FSH Society, the world’s largest research-focused patient organization for facioscapulohumeral muscular dystrophy (FSHD), announced the launch of its national chapter development program to provide the opportunity to fund more research, connect with more patients, and speed up progress toward treatments and a cure for FSHD. FSHD is among the most prevalent of the nine … Read more of “FSH Society launches national chapter program” »

Gene editing takes big step forward in Duchenne dog model

Researchers at the University of Texas Southwestern Medical Center in Dallas announced that they have made a significant advance in demonstrating the possibility of using CRISPR gene editing technology as a therapy for Duchenne muscular dystrophy (DMD). The technology was designed to “patch” a mutation in the dystrophin gene, which is defective in DMD, and … Read more of “Gene editing takes big step forward in Duchenne dog model” »

Facio broadens portfolio of potential drug development candidates

Reposted from Facio Therapies, Leiden, The Netherlands – August 30, 2018  Facio Therapies announced today that it has selected a second series of potential candidates for FSHD drug development. This series encompasses a family of novel small-molecule compounds that repress DUX4 by engaging a molecular target that differs from the target of Facio’s first series … Read more of “Facio broadens portfolio of potential drug development candidates” »