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 . . .
Justin Moy’s future is brimming with potential. The 17-year-old Concord, Mass., native is currently a senior at Concord-Carlisle High School, where he enjoys science and participating in the school choir. Although he hasn’t yet figured out what college he’s going to attend, he knows what he wants to do once he gets there. “I want . . .
When Don and Leeann Fortenberry of Waco, Texas, learned their daughter Faith had spinal muscular atrophy (SMA) type 2, they were motivated to take action. Although the diagnosis came as a shock, Don and Leeann were determined to give Faith the best life possible while doing everything in their power to raise awareness for those . . .
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.
One year ago, if you would have asked me to say the words Duchenne muscular dystrophy, I couldn’t. It was too new, too fresh, too difficult of a reality to process. One year ago I fell to my knees crying and physically shaking with fear, anxiety and anger as I hung up the phone with . . .
In January, MDA launched our first-ever patient experience survey. This tool is designed to provide a statistically accurate snapshot of our community — the people who live with or love someone with neuromuscular diseases – and their needs.