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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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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 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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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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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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– 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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Genethon reported positive results from its AAV-microdystrophin treatment trial in Europe.  The three participants receiving the study’s higher dose (3×1013vg/kg) showed that an average of 54% of muscle fibers were producing microdystrophin, and individuals demonstrated a reduced level of creatine kinase, a marker of muscle damage.  Genethon said it is planning a pivotal trial in…

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REGENXBIO has announced it initiated enrollment in a new cohort of patients ages 1-3 in its Phase I/II AFFINITY DUCHENNE® trial. The trial is evaluating the safety and efficacy of RGX-202, an investigational gene therapy that utilizes a novel adeno-associated virus (AAV8) to transport a shortened version of the dystrophin gene (micro-dystrophin) that may provide…

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