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Early preventive heart treatment extends survival in DMD: Study
Preventive treatment with standard heart medications — when given before the onset of cardiac troubles — extends survival among males with Duchenne muscular dystrophy (DMD), according to data from the U.S.-based Muscular Dystrophy Surveillance, Tracking and Research Network, known as MD STARnet. However, despite finding such benefits with prophylactic, or preventive, heart treatments, a team…
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Simply Stated: Updates in Pompe Disease and Other Glycogen Storage Diseases
Glycogen storage diseases (GSDs) are a group of rare inherited conditions that occur when the body is not able to use or store glycogen properly. People with GSDs may experience frequent low blood sugar (hypoglycemia), muscle weakness, and liver damage. Glycogen is processed by many different enzymes in the body, and defects in some of…
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MDA’s Development Grants Support Promising Researchers Entering the Neuromuscular Field
Since its founding 75 years ago, MDA has been the nation’s largest nonprofit supporter of neuromuscular disease research. Every year, MDA awards grants to support promising research in the field. Over the years, MDA has supported more than 9,000 scientific investigators, and about 2,500 have received Development Grants. These grants have a specific purpose: nurturing…
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FSHD progression requires me to lighten my music rig
I’ve played music since I was 5 years old. My first professional playing job was as an accordionist in a Polish polka band when I was 13. My accordion at the time was a full-size Excelsior 120 bass model that weighed about 35 pounds. My professional playing career took a lot of turns over the…
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Kicking off fresh creative ventures through social media
Despite the significant health challenges I face as a person living with Duchenne muscular dystrophy, I’ve come to realize that creativity can thrive in the most unexpected places. One such place is social media. My girlfriend, Amanda Yip, and I recently embarked on a short-term project with Blind Mice Media, a company dedicated to enhancing…
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ENTR-601-44 trial for DMD adults amenable to exon 44 skipping OK’d
The U.S. Food and Drug Administration (FDA) has given Entrada Therapeutics permission to launch a Phase 1b clinical trial to test ENTR-601-44, the company’s experimental exon-skipping therapy, in adults with Duchenne muscular dystrophy (DMD) who carry a mutation that’s amenable to exon 44 skipping. Entrada had sought the FDA’s OK to start the trial in…
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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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How an emergency medical plan can benefit those of us with MD
“ICE” is a widely used acronym for “in case of emergency.” In today’s fast-paced world, many people have ICE contacts on their phones and folders on their computers that tell others who to call in the event of an emergency. Those of us living with chronic illness can and should have clear and concrete steps…
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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…
