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Simpler, Safer Transfers: Helpful Tips and Useful Equipment
Theresa Berner is an occupational therapist. Transferring from one spot to another, such as from a bed to a wheelchair and from a wheelchair to a toilet, can be a daily challenge for those living with neuromuscular diseases. Whether at home or out and about, being able to transfer easily, safely, and comfortably is a…
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With summer’s hardships come adventures in appreciation
Summer is a great time to be outside, travel, attend neighborhood gatherings, and spend time with people you love. But those of us who live our adult lives with chronic illness face challenges in this season, as well as year-round. Still, there are many ways for us to enjoy the warm weather and continue to…
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Webinar Recording: Edgewise Therapeutics – Update on Sevasemten Program in Becker and Duchenne
Edgewise Therapeutics recently joined PPMD for a community webinar for an update on the company’s sevasemten program in Duchenne and Becker. The Edgewise team reviewed new Open-label Extension MESA data in Becker, as well as interim data from the ongoing LYNX and FOX Phase 2 studies. Watch the recording The post Webinar Recording: Edgewise Therapeutics…
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Ifetroban continues to show cardiac protection in Phase 2 DMD trial
Ifetroban, Cumberland Pharmaceuticals’ investigational oral therapy, improved heart function and reduced cardiac damage biomarkers in people with Duchenne muscular dystrophy (DMD), according to new trial data. These findings from the Phase 2 FIGHT DMD trial (NCT03340675) were presented at the Parent Project Muscular Dystrophy (PPMD) annual conference, held recently in Las Vegas. Long-term safety and…
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Craft Your Story
Crafting Your Story for Awareness, Connection, and Change Webinar. Watch HERE The post Craft Your Story appeared first on CureDuchenne.
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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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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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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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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…
