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US leadership emphasizes support for rare disease drug development
CureDuchenne is pleased that one of the main themes at the recent US FDA Cell and Gene Therapy Roundtable was preserving incentives and leveraging regulatory flexibility and innovation to remove obstacles for rare disease treatment. Read more here The post US leadership emphasizes support for rare disease drug development appeared first on CureDuchenne.
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Quest Podcast: Invisible People: Making the Rare Seen
In this Quest Podcast episode, we chat with a former pharmacist turned singer/songwriter who lives with Generalized Myasthenia Gravis. Dania Quill has devoted her time and expertise to create inclusive spaces for those with disabilities and deliver advice, inspire action, and make us feel closer through song while sharing stories of resilience and positivity. While…
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REGENXBIO Shares Positive Functional Data from Phase I/II AFFINITY DUCHENNE Trial of RGX-202
Today, REGENXBIO announced new data from its ongoing Phase I/II AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne muscular dystrophy. RGX-202 is designed to deliver microdystrophin via AAV8 through a one-time IV infusion. The newly released data comes from boys treated at the higher of two doses (2×1014…
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PPMD’s Certified Care Center Program
PPMD continues to advocate to ensure families navigating a diagnosis of Duchenne and Becker muscular dystrophy have access to optimal care for their loved ones. The Certified Duchenne Care Center Program (CDCC Program), a unique program of PPMD, aims to ensure all individuals with Duchenne and Becker have access to high quality, comprehensive, and coordinated…
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FDA grants orphan drug status to ISX9-CPC for heart issues in DMD
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS Heart’s experimental stem cell therapy for heart problems in Duchenne muscular dystrophy (DMD), the company announced in an email sent to Muscular Dystrophy News Today. This FDA designation aims to accelerate the development of treatments for rare diseases, like DMD,…
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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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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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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…
