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FDA OKs expansion of DMD stem cell trial, allows 30 more boys
The U.S. Food and Drug Administration (FDA) has allowed the enrollment of up to 30 additional boys with Duchenne muscular dystrophy (DMD) in a clinical study testing an experimental stem cell therapy. The Phase 2 trial (NCT06579352) testing the treatment from Signature Biologics has enrolled five boys, ages 5 to 10, at two sites in…
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Building a Stronger Therapeutic Pipeline for Duchenne
CureDuchenne was founded 25 years ago, and during that time we have witnessed and participated in the approval of multiple FDA-approved therapies, dozens of clinical trials, and an expanding pipeline of investigational treatments. But there is still no cure for Duchenne, and important challenges remain to be solved. For families living with Duchenne, every scientific breakthrough brings hope, and every clinical trial represents another possibility. Real progress requires the combined efforts of researchers,…
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MDA Ambassador Guest Blog: Accessible Air Travel is About More Than Getting from Point A to Point B
James (Jimmy) Grammig is a 23-year-old from Tampa, Florida. He was diagnosed with limb-girdle muscular dystrophy (LGMD) and has used a wheelchair since he was about 13 years old. He graduated from the University of Notre Dame with a degree in management consulting. Jimmy spent a gap year working as a volunteer teacher in his…
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New Myotubular Myopathy Clinical Trial May Lead to Safer Gene Therapy
At 10 months old, Stevie touched his feet for the first time. Before this moment, he did not exhibit any of the typical motor skills for his age. He could not sit up on his own, crawl, or pick up toys. Stevie in 2018 Stevie was born with myotubular myopathy (MTM, also called X-linked myotubular…
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Capricor Therapeutics recently shared an update on the FDA review
Capricor Therapeutics recently shared an update on the FDA review of their investigational therapy, deramiocel. The company shared that the Biologics License Application (BLA) for deramiocel remains under FDA review as it prepares an amendment seeking a narrower DMD indication focused on upper-limb skeletal muscle function. Capricor said the FDA has indicated it will review…
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Clinical Research Alert: Clinical Study for Children and Teenagers with SMA
Researchers at argenx are conducting a phase 2 clinical trial (SPARKLE) for children and teenagers with spinal muscular atrophy (SMA). The study is designed to determine the appropriate dose of the investigational drug adimanebart (ARGX-119) and to evaluate its safety and effectiveness as a potential treatment for SMA. Researchers will also examine how the drug…
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A vacation with close friends was a perfect way to end the summer
My family recently spent a week in Indiana with friends at a cabin on a lake, and it was so good for us. Our two families met more than a decade ago when our sons were all little boys, and we have remained friends throughout our separate journeys with Duchenne muscular dystrophy (DMD). My husband…
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Update on the FDA’s Review of Deramiocel: Continuing the Path Forward
Today, Capricor Therapeutics shared an important update on the FDA’s review of the Biologics License Application (BLA) for Deramiocel, the company’s cell therapy for the treatment of Duchenne muscular dystrophy-associated cardiomyopathy. Capricor has indicated that the company plans to submit an amendment to the FDA that will include additional data and analyses to support the…
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US developer gearing up for first-in-human trial of Duchenne cell therapy
A U.S. biopharmaceutical company is gearing up to launch its first clinical trial of a novel cell therapy designed to restore lost muscle cells in people with Duchenne muscular dystrophy (DMD). Texas-based IPS Heart announced that it is preparing an investigational new drug application or IND — a formal request to the U.S. Food and Drug…
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3,500-year-old skeletons challenge what scientists thought they knew about syphilis
Ancient remains from Vietnam suggest diseases like yaws may have been passed from mother to child thousands of years ago, a form of transmission often assumed to indicate syphilis. The discovery could overturn how scientists interpret ancient infections and complicate the long-running mystery of syphilis’s origins.
