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Regenxbio reports positive interim data from Phase 1/2 microdystrophin gene therapy trial
Regenxbio shared positive data from the AFFINITY DUCHENNE trial of RGX-202. In addition to robust microdystrophin expression, Regenxbio reported functional benefits at 9 and 12 months after treatment for all of the first 5 participants receiving the pivotal dose (2E14 GC/kg), aged 6-12 years at dosing. You can learn more here and during a webinar [REGISTER HERE], CureDuchenne…
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Building empathy for Duchenne experiences through theater
Last Thursday, I participated in a workshop that used experiential theater to examine healthcare conversations. With a sense of anticipation and hope, I stepped into the unknown, eager to gain insights that could enrich my advocacy for Duchenne muscular dystrophy (DMD), which I have. The workshop, held by the SingHealth Patient Advocacy Network (SPAN), is…
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Pepgen pulls plug on exon-skipping therapy for Duchenne MD
Following lackluster data from a Phase 2 clinical trial, Pepgen will discontinue developing PGN-ED051, its experimental exon 51-skipping therapy for Duchenne muscular dystrophy (DMD). The company said it will wind down all DMD-related research and development activities, focusing instead on an investigational therapy for myotonic dystrophy type 1 (DM1) that’s in clinical testing. “As we wind…
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MDA Ambassador Guest Blog: How My MG Journey Has Shaped Who I Am Today
Courtney B. is a passionate advocate for living life to the fullest despite the challenges that come with chronic illness. A high school senior from Indiana, she is an early graduate, a cheerleader, a lifeguard, and a law intern — all while managing the complexities of multiple health conditions, including myasthenia gravis. After being told…
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Steroids and Stress Dosing: Understanding the Updated PJ Nicholoff Steroid Protocol
Last week, PPMD brought together leading experts to address steroids, stress dosing and the updated PJ Nicholoff Steroid Protocol. Drs. Aravindhan Veerapandiyan, David Weber, and Anne Marie Sbrocchi shared valuable insights on the management of steroids, importance of stress dosing, and helping families navigate the effects of long-term corticosteroid treatment with the PJ Nicholoff Steroid…
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Why those of us with FSHD feel like we’re always in transition
During a recent facioscapulohumeral muscular dystrophy (FSHD) wellness group meeting via Zoom, we each shared how we were feeling about our disease journey. One participant said, “I feel like I’m always in transition.” I can relate to that. With FSHD, the need to adapt seems never-ending, a topic that has frequented my thoughts over the…
