By June Kinoshita, Director of Research and Patient Engagement “Positive benefit/risk supports losmapimod’s potential to be a transformative therapy for the treatment of FSHD”—Fulcrum Therapeutics Fulcrum Therapeutics announced today that losmapimod, its experimental therapy for facioscapulohumeral muscular dystrophy, produced statistically significant improvements in function and decreased fatty infiltration of muscle in its ReDUX4 clinical trial…. Read More »
ReDUX4 trial result exceeds expectations
COVID-19 Vaccine Q&A
This Q&A is based on the December 9 webinar with Rabi Tawil, MD, and Ghinwa Dumyati, MD, from the University of Rochester Medical Center, and the January 15 webinar hosted by the National Organization of Rare Diseases with Stephen Hahn, MD, FDA commissioner; Peter Marks, MD PhD, director of the FDA’s Center on Biologics Evaluation… Read More »
FSHD drug development pipeline
Growing numbers of companies and academic laboratories are pressing forward with early-stage drug development efforts. This chart shows how far various candidate anti-DUX4 drugs have progressed along the path to FDA approval. DUX4 is considered a key gene causing FSHD. The process begins with finding a “target,” or biological disease mechanism, that can be modified… Read More »
Anyone can MOVE for FSHD research
What’s so great about the MOVE study? A lot, if you are research geeks like us or care about getting treatments to patients sooner. MOVE is what’s called an “observational” or “natural history” study of people with facioscapulohumeral muscular dystrophy (FSHD) that evaluates how symptoms and abilities change over a period of time. It is… Read More »
Ayurvedic medicine for FSHD – My story
by Kulangara Sivadas, Foothill Ranch, California Editor’s note: This story is one person’s anecdotal experience. He has asked Western-trained physicians to comment on it. Their remarks are added at the end of the story. In addition, we recommend you read this Johns Hopkins Medicine page about Ayruveda. For more than three years I’ve been contemplating… Read More »
2021 Best Poster Award goes to Alec DeSimone
Alec DeSimone, PhD, currently a postdoctoral researcher at Yale School of Medicine, was the winner of the Best Poster Award at the 2021 International Research Congress, held virtually on June 24-25. The title of his poster “A live-cell drug screening platform for FSHD therapeutics,” co-authored with Drs. Angela Lek and Monkol Lek, describes a cost-effective,… Read More »
Giorgio Tasca wins 2021 Young Investigator Award
The 2021 FSHD Society Young Investigator Award at the International Research Congress was awarded to Giorgio Tasca, MD PhD, a neurologist at the Fondazione Policlinico Universitario in Rome, Italy. Dr. Tasca received his medical degree (MD) with honors at the Catholic University in Rome, Italy in 2005 and his PhD in Neuroscience at the same… Read More »