June 2024

Parent Project Muscular Dystrophy (PPMD) held its first conference in 1994, in Orlando, Florida. A year later, at its meeting in Pittsburgh, fewer than two dozen people were in attendance, just a small group of parents of children with Duchenne muscular dystrophy (DMD) and a few scientists interested in the disease. It was at that…

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“I didn’t think you’d jump with us,” said my daughter Mary, 9, breathing heavily as she bounced from trampoline to trampoline. It’s true; I’d never taken her or her little sister Callie, 2, to the trampoline park before. But we do fun things, I thought. After thinking some more, however, I now realize that —…

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The Parent Project Muscular Dystrophy (PPMD) 30th annual conference kicks off today, offering families affected by Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) the chance to “celebrate three decades of progress, community, and resilience.” The PPMD conference, which runs through Saturday, is taking place in Orlando, Florida. The cost to attend is $30…

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Capricor Therapeutics today announced additional positive 3-year safety and efficacy results from the company’s ongoing HOPE-2 open label extension (OLE) study of deramiocel (CAP-1002) for the treatment of Duchenne. CAP-1002 is the company’s novel cell therapy. Data from the HOPE-2 OLE study demonstrated improvements in multiple cardiac measures, including left ventricular ejection fraction (LVEF), as…

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Capricor Therapeutics will soon meet with the U.S. Food and Drug Administration (FDA) to discuss plans for an application seeking approval of the company’s CAP-1002 cell therapy, now named deramiocel, as a treatment for Duchenne muscular dystrophy (DMD). The FDA has scheduled the pre-biologics license application (BLA) meeting for the third quarter of this year,…

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A Phase 3 trial testing an experimental therapy for limb-girdle muscular dystrophy type 2i (LGMD2i) has surpassed patient enrollment for a planned interim analysis, the therapy’s developer BridgeBio Pharma has announced. The Phase 3 FORTIFY trial (NCT05775848) is evaluating the long-term safety and efficacy of BBP-418 in adolescents and adults, ages 12-60, with LGMD2i. The interim…

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Last week, my wife, Wendy, and I finally got around to starting the process of purchasing a new mattress. We realized when we moved to Pittsburgh in January 2023 that we needed to replace our mattress, which has served us well for many more years than the sleep experts recommend. It’s been so long that…

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Leading into PPMD’s 30th Annual Conference, PPMD is excited to host a professional meeting focusing on Becker muscular dystrophy care. Over the course of two days, attendees from across subspecialties, academic medical centers, and industry partners will explore a number of key issues specific to the care of individuals with Becker, including: What we know…

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– ENTR-601-44 was well-tolerated in healthy volunteers with no serious adverse events, no drug-related adverse events and no clinically significant changes or trends noted in vital signs, ECGs, physical exams or laboratory assessments – – ENTR-601-44 demonstrated significant plasma concentration, muscle concentration and exon skipping, at levels that suggest the potential for a clinically meaningful starting…

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Company to begin enrollment of patients aged 1-3 years Expects data from younger cohort to be part of pivotal plans and BLA filing for broad label End-of-Phase II meeting with FDA scheduled for late July to finalize pivotal program design Based on recent commercial landscape, confirmed accelerated approval pathway remains available given ongoing unmet need and RGX-202…

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