Last week, I had the opportunity to attend the 2018 MDA Clinical Conference in Arlington, Va., to write about it from the patient’s perspective. I am always excited to attend conferences that bring together stakeholders in the neuromuscular disease community. I love meeting fellow patients, and I especially love learning about the latest scientific and . . .
Solid Biosciences has announced that the first patient to be treated in its phase 1/2 clinical trial to test SGT-001, the company’s experimental gene therapy treatment for DMD, experienced a serious safety issue that was not among what was predicted based on what is known about the therapy — known as a Suspected Unexpected Serious . . .
Researchers are looking for children and adults with congenital myasthenic syndrome (CMS) to participate in a phase 3 study being conducted by Catalyst Pharmaceuticals to test the experimental drug amifampridine phosphate (brand name Firdapse).
MDA’s 2018 Clinical Conference is the preeminent gathering of MDA Care Center health care providers and other medical and scientific experts from across the country designed to enhance the communication of new medical advances and information relating to the delivery of best-in-class care for individuals with neuromuscular disease.
In MDA’s pursuit to find treatments and cures for neuromuscular diseases we awarded four grants to researchers studying SBMA and SMA in 2017. Check out their research here:
During 2017 we were honored to provide four research grants and one development grant to individuals looking to accelerate treatments and cures for FSHD and FA. Learn more about their work:
Throughout 2017, MDA awarded five research grants and a clinical research training fellowship to individuals dedicated to finding treatments and cures for myotonic dystrophy (DM).
Acceleron Pharma announced encouraging preliminary results from its phase 2 clinical trial to test the experimental drug ACE-083 in FSHD.
In the last year MDA awarded a number of research, development and venture philanthropy grants to individuals dedicated to finding treatments and cures for ALS.
Since 1986, when MDA-funded researchers identified the gene that, when flawed, leads to Duchenne muscular dystrophy (DMD), scientists have been actively pursuing treatments and cures. In the past 12 months, MDA has awarded research grants to the following individuals who are building on the strong foundation of previous research and discovery to better understand — and someday cure — the diseases.
