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Roche halts development of satralizumab for DMD bone health
Roche has decided to stop developing satralizumab for bone health in Duchenne muscular dystrophy (DMD), the company announced in a community letter. Patients already enrolled in the SHIELD DMD Phase 2 trial (NCT06450639) may continue on the study until the six-month bone mineral density collection, expected in the second half of the year. Those currently…
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Quest Podcast: Fashion for Every Body: Izzy Camilleri on Style, Function, and Inclusion
In this Quest Podcast episode, we chat with internationally recognized fashion designer Izzy Camilleri, a true pioneer in adaptive fashion. She shares how her successful career in high-end fashion took a transformative turn when she began designing clothing for people with disabilities and partnered with Silverts—work that helped ignite today’s adaptive fashion movement. Izzy shares…
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Progress Now: Research Updates and Breakthroughs
Amyotrophic lateral sclerosis (ALS) Phase 1 Clinical Trial: Recruiting This study, called LUMINA, is testing an investigational therapy, called AMX0114, in adults with ALS. The main goal is to learn about safety and how well the treatment is tolerated. The study will also look for early signs that the therapy may help people with ALS.…
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An essay on choosing hope in life with a progressive, degenerative disease
I hate muscular dystrophy. In my darker moments, I want to scream into the void of the universe — until I remember that I can’t scream anymore. In crowded spaces, others often can’t hear me, so I remain quiet. This goes against every fiber of my being; everyone who knows me knows how much I…
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Young Leader Living with Muscular Dystrophy Champions Aiming High and Setting Goals
Harvard graduate Caroline LeMay’s education helped pave the way on her quest for success. Now, she is dedicating her career to increasing access to education for others. Caroline LeMay, her husband, and their dogs The 27-year-old already has an impressive résumé, with positions as a financial analyst for J.P. Morgan Markets, a special advisor to…
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A Guide to RNA-Targeted Therapies
A Guide to RNA-Targeted Therapies The post A Guide to RNA-Targeted Therapies appeared first on Quest | Muscular Dystrophy Association.
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RNA Therapies Offer Keys to Treating Genetic Neuromuscular Diseases
While DNA holds our genetic code, RNA plays a vital role in gene expression. Researchers are discovering new ways to use RNA to correct genetic changes that cause diseases. For example, in type 1 myotonic dystrophy (DM1), a variety of symptoms all stem from a single source: incorrectly produced, toxic proteins. As bricks are to…
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Understanding Thymidine Kinase 2 Deficiency
Thymidine kinase 2 deficiency (TK2d) is a life-threatening form of mitochondrial myopathy. These diseases affect mitochondria — the energy factories of our cells — leading to muscular problems. TK2d is very rare, affecting fewer than 2 per 1 million people worldwide. Yet in a milestone for mitochondrial myopathies, the US Food and Drug Administration (FDA)…
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Expert Tips for Handling an Insurance Claim Denial for Gene Therapy
When Alison Joseph and William Small’s two youngest sons were diagnosed with Duchenne muscular dystrophy (DMD) in 2017, they were told there were no treatment options. The Small Family “Because of their specific mutation, they didn’t qualify for exon-skipping or gene therapy trials. We were just doing standard-of-care steroids, hoping for something new,” Alison says.…
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Considering a Clinical Trial? 4 Things to Know Before You Enroll
For people living with neuromuscular diseases, few things bring more hope than progress in research, whether it leads to a new therapy or a deeper understanding of a diagnosis. Each advancement represents years of scientific work — and the dedication of those who volunteer for clinical trials. While exciting, enrolling in a study is a…
