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Entrada Therapeutics announces the FDA has removed the clinical hold on ENTR-601-44, their experimental therapeutic designed to skip exon 44
As an early funder of Entrada Therapeutics, we are pleased to share that the FDA has removed the clinical hold on ENTR-601-44, the experimental therapeutic designed for individuals with Duchenne amenable to skipping exon 44. ENTR-601-44 is a phosphorodiamidate morpholino oligomer (PMO) conjugated to a proprietary Endosomal Escape Vehicle (EEV™), which aims to facilitate delivery…
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Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44
Entrada Therapeutics, Inc. has announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on ENTR-601-44 and provided authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose clinical study of ENTR-601-44. ENTR-601-44 is the company’s proprietary Endosomal Escape Vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer (PMO), for the potential treatment of individuals…
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Solid Biosciences Reports Positive Initial Clinical Data
Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003 Solid Biosciences, announced positive initial results from INSPIRE DUCHENNE, their next-generation gene therapy clinical study of SGT-003. Interim 90-day biopsy data reported in the first three participants showed an average microdystrophin expression of 110%, as measured by western blot, and improvements in…
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Caregiving sometimes feels like walking a tightrope
My heart is walking a tightrope. I’m a 45-year-old wife to Jason and mother to our seven children: Lexi, 23, Max, 19, Chance, 17, Rowen, 16, Charlie, 14, Mary, 10, and Callie, 3. Three of our children, Max, Rowen, and Charlie, are living with Duchenne muscular dystrophy (DMD). This year, my life has changed in…
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Life with Lily: Finding My Voice
Hello everyone! I’m so excited to launch my quarterly blog series as your 2025 National Ambassador! This first “Life with Lily” post is deeply personal, a reflection on my journey with Charcot-Marie-Tooth (CMT) disease and an exploration of the nuanced realities of the disability experience. I want to share my story, not just as a…
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2024 MDA Advocacy Collaboration Grantees are Working Towards Change
Advocacy for the neuromuscular disease community is just one of the key ways MDA works to support the neuromuscular disease community. To expand our impact and undertake projects of importance to the community, we strive to foster partnerships that advance the ability of people with neuromuscular diseases to live longer, healthier, and more empowered lives.…
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MDA’s Origin Story: Grassroots Legacy
As we celebrate MDA’s 75th anniversary, you may be asking, how did this all begin? It’s no surprise that MDA started as a grassroots organization. From the earliest days, our mission has been shaped by the stories and needs of people living with neuromuscular diseases and their families. Founded in 1950 MDA began in 1950…
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First Semester in a New York Minute
Jonathan Lengel is an actor, singer, and performer best known for his role as Archie in Netflix’s Broadway Adaptation of 13: the Musical. Jonathan lives with a rare form of congenital muscular dystrophy. An MDA Ambassador since 2019, Jonathan received an inaugural MDA Scholarship in 2024. He is currently pursuing a double major in Digital…
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Hoyer Lifts and Beyond: Choosing the Right Lift Device
For those living with limited mobility from a neuromuscular disease or other disability, assisted transfers can be a big part of daily life. Many people who can’t stand and pivot or don’t have the upper body strength to use a sliding board independently require a transfer lift or lift system. These devices generally have a…
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Patients with more siblings report being better prepared for adulthood
Adolescents and young adults with Duchenne (DMD) or Becker muscular dystrophy (BMD) who have more siblings typically report being better prepared to transition to adult life, a new study has found. Findings show people with DMD or BMD had the most difficulty with activities of daily living, education, and employment when navigating adulthood, and fewer…
