Tricyclo-DNA (tcDNA), a class of therapeutics known as antisense oligonucleotides (AONs), has shown an important degree of effectiveness and a promising safety profile in a preclinical study with mice. The goal of the study is to test tcDNA’s potential use in treating Duchenne Muscular Dystrophy (DMD). The study, “Efficacy and…
In this video from Muscular Dystrophy UK, patients, caregivers and medical professionals talk about some of the best practices that should be adopted when it comes to the treatment and management of muscle-wasting diseases such as muscular dystrophy (MD) and spinal muscular atrophy (SMA). They explain how many…
The newly approved Duchenne muscular dystrophy therapy Exondys 51 (eteplirsen) stays in immune cells several days after leaving the blood, continuing to help regenerate muscle fibers, according to a study in mice. Researchers at the Children’s National Health System in the Washington area said the findings on immune cells known as…
The biotechnology company Capricor Therapeutics recently presented data that support the use of its peripheral intravenous delivery method in the upcoming HOPE-2 clinical trial testing the investigational agent CAP-1002 as a potential therapy for Duchenne muscular dystrophy (DMD). The data were presented during a poster session at the Alliance…
A new version of the CRISPR-Cas9 gene-editing technology called CRISPR-Gold has successfully restored the correct sequence of the dystrophin gene in a mouse model of Duchenne muscular dystrophy (DMD), a new study revealed. Researchers found that an injection of CRISPR-Gold into DMD mice led to an 18-times-higher correction rate and…
Mice with Duchenne muscular dystrophy compensate for the weakness in their breathing muscles by sending stronger signals to the nerves controlling the diaphragm, according to scientists at the University College Cork in Ireland. Now it is important to examine whether the same mechanisms exist in human Duchenne patients, researchers…
Mattie Stepanek’s story is well-known in the MD community. Mattie was the youngest of four children, all of whom died in childhood from complications due to the genetic neuromuscular disorder, dysautonomic mitochondrial myopathy. MORE: Musclar dystrophy quote: Mattie Stepanek Mattie touched the hearts of millions of Americans and was a…
In this episode of Muscle Owl Talks, host Peter Duffy chats with Jon Hastie, a Duchenne muscular dystrophy patient who made the film A Life Worth Living. Jon talks about the film and his charity DMD Pathfinders and what it’s like living with Duchenne as an adult.
Orphan drug development, rising healthcare costs, patient assistance programs and controversial right-to-try legislation are of paramount interest to the parents and caregivers of boys with Duchenne muscular dystrophy (DMD). “One of the chief challenges of 21st Century pediatric medicine is our continued inability to provide more help to children born with…
Resolaris (ATYR1940) improved the muscle strength of nearly two-thirds of adolescents and young adults with early-onset facioscapulohumeral muscular dystrophy (FSHD), according to final results of a Phase 1/2 clinical trial. The therapy’s maker, aTyr Pharma, presented the findings at the 22nd International Annual Congress of the World Muscle Society (WMS) in Saint…
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