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Simply Stated: MG Therapeutic Landscape
Myasthenia gravis (MG) is a chronic neuromuscular disease characterized by muscle weakness that worsens after activity and improves after rest. Most people with MG experience one of two forms, ocular or generalized. In ocular MG, weakness is limited to the muscles that move the eyes and eyelids. In generalized MG (gMG), weakness may affect eye…
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Clinical Research Alert: Phase 3 Study of RGX-202 in Boys with DMD
REGENXBIO Inc. is enrolling boys living with Duchenne muscular dystrophy (DMD) to participate in a phase 3 clinical trial to evaluate the safety, tolerability and efficacy of the investigational gene therapy RGX-202 to treat DMD. RGX-202 is designed to produce a novel microdystrophin protein to replace the missing dystrophin protein in the muscles of males…
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Arizona Becomes the Latest State to Require Newborn Screening for Duchenne
We are proud to share some important news for our community: Arizona has officially joined the growing list of states requiring newborn screening for Duchenne! Thanks to the tireless efforts of passionate advocates, dedicated families, and strong support from our community, the Duchenne newborn screening bill was included in Arizona’s state budget. On Friday, June…
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Accepting help is difficult for me as a mom and a caregiver
Late afternoon sunlight drifts into the house, brightening the blue paint on the walls. The room feels cheery and welcoming. Wicker baskets hold soft blankets, pillows cushion the couch, and dogs are sleeping on the rugs. I love walking into this room, and every room, in my home, which I have carefully curated to provide…
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MDA Ambassadors Share Tips for Flying with Power Wheelchairs and Medical Equipment
Air travel, even in the best circumstances, can be a little stressful. Flying with a power wheelchair or other mobility or medical equipment adds complications and hurdles to getting on an airplane. Lyza Weisman loves to travel to new places. But for many people with neuromuscular diseases, the benefits of travel are worth the challenges.…
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Precision’s gene-editing therapy gets FDA’s rare pediatric disease status
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Precision Biosciences. The designation provides companies with extra incentives to develop experimental therapies to treat rare diseases that mainly affect children. “The receipt of Rare Pediatric Disease…
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Edgewise Therapeutics Reports Positive Results on Sevasemten Program
Edgewise Therapeutics, which received early funding from CureDuchenne, has reported positive results in its sevasemten program for Becker and Duchenne muscular dystrophies. New open-label data in Becker from the MESA trial demonstrated sustained disease stabilization up to three years in participants, reinforcing prior clinical findings from the ARCH and CANYON trials. Edgewise also reported encouraging topline data from…
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Edgewise Therapeutics Shares Positive Results on Sevasemten Program for Becker and Duchenne
Edgewise Therapeutics, Inc., has announced positive results in the company’s sevasemten program for both Becker and Duchenne. These data include results from: MESA, an open label extension trial of participants with Becker who were previously enrolled in the ARCH trial, or completed the CANYON, GRAND CANYON, or DUNE trials Phase 2 Duchenne trials, LYNX and…
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An odd dream made me wonder how my disability affects others
I’m an early riser. As I mentioned in a past column, I like to wake up early, get dressed, make coffee, and start the day with some quiet prayer and devotion time. However, I’m also a very sound sleeper, with some help from melatonin, sleep medication, and a comfy mattress. From the time I fall…
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FDA advisory committee meeting for deramiocel in DMD set for July
The U.S. Food and Drug Administration (FDA) is due to convene an advisory committee meeting next month to review the data on deramiocel, an experimental cell therapy up for approval to treat heart disease in people with Duchenne muscular dystrophy (DMD). The FDA has informed Capricor Therapeutics the meeting should happen July 30, though Capricor…
