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99 results for "muscular dystrophy"
Scientists at Carnegie Mellon University have discovered a precise way to target toxic RNA that causes myotonic dystrophy type 1, the most common adult-onset muscular dystrophy affecting 1 in 2,300 people worldwide. The breakthrough could lead to treatments with fewer side effects for this currently incurable disease and other devastating genetic disorders.
Scientists at Carnegie Mellon University have discovered a breakthrough approach to target the root cause of myotonic dystrophy type 1, the most common form of adult muscular dystrophy. The precision therapy could finally offer hope to patients with a disease that currently has no effective treatment.
Scientists discovered that a chromosome-protecting protein also helps muscle stem cells maintain their ability to heal injuries, offering new hope for treating muscular dystrophy. Without this protein, damaged muscle turns to fat and scar tissue instead of rebuilding properly.
A breakthrough study reveals why a new treatment for Duchenne muscular dystrophy helps young boys gain strength without stunting their growth, unlike traditional steroids. Blood tests show the safer drug preserves bone and cartilage health while delivering the same muscle benefits.
A University of Nevada scientist has discovered a promising drug that could help protect muscles in patients with deadly Duchenne muscular dystrophy. After 20 years of research, Dean Burkin's team is preparing to test their breakthrough therapy in human trials.
A new gene therapy for Duchenne muscular dystrophy met its main goal in trials, bringing hope to families facing this devastating disease. The treatment successfully helped 28 of 30 patients produce a crucial muscle protein that could slow disease progression.
A gene therapy called ELEVIDYS is helping young boys with Duchenne muscular dystrophy maintain their ability to walk, run, and stand years after a single treatment. Three years after receiving the therapy, treated children showed 70% less decline in muscle function compared to untreated children.
A rare muscle disease that once offered families almost no hope now has multiple new treatments helping kids stay stronger longer. Gene therapies and innovative medications are changing what's possible for children with Duchenne Muscular Dystrophy.
University of South Florida researchers discovered an experimental drug that protects heart function in Duchenne muscular dystrophy, potentially transforming care for boys with this fatal genetic disease. The treatment reduced heart damage and scarring in preclinical studies, offering new hope for families facing this devastating condition.
Scientists at Penn Medicine discovered that a chromosome-protecting protein also helps muscle stem cells maintain their ability to repair damaged tissue. Without this protein, injured muscles turn to fat and scar tissue instead of healing.
Scientists in Japan have discovered that simple iron supplements can significantly improve muscle strength in mice with a rare form of muscular dystrophy, offering hope for thousands of patients with no current treatment options. The breakthrough works by correcting iron imbalances in muscle tissue, not by fixing the underlying genetic cause.
A groundbreaking RNA therapy just succeeded in early trials for a rare muscular dystrophy affecting thousands, and it could become the first treatment that actually modifies the disease's progression. Novartis's del-brax showed clear signs of reducing muscle damage in patients with facioscapulohumeral muscular dystrophy.
The Muscular Dystrophy Association's annual conference brings together scientists and advocates to share breakthroughs that could change lives. Registration is now open for the March 2027 event, where cutting-edge research meets real-world hope.
After a difficult year, biotech company Sarepta Therapeutics is bouncing back with early clinical trial results showing two new muscular dystrophy treatments appear safe and effective. The promising data offers hope for patients with rare muscle-wasting diseases who desperately need new treatment options.
A groundbreaking treatment that switches off a faulty gene is showing early success in 12 people with a rare form of muscular dystrophy. Instead of losing muscle as expected, patients are gaining strength.
A nonprofit and patient foundation just committed $7.65 million to develop a gene therapy for a rare muscular dystrophy with no current treatments. The partnership will take the therapy from initial design all the way to human clinical trials.
Boys with Duchenne muscular dystrophy are showing sustained improvements three years after receiving Elevidys gene therapy, demonstrating the treatment can slow progression of this muscle-wasting disease. The results offer hope to families facing a condition that typically causes rapid physical decline around age 9.
Pharmaceutical company Roche announced a new clinical trial that could bring a life-changing gene therapy for Duchenne muscular dystrophy to young patients across Europe. The move comes after extensive feedback from families and regulators who want to see this treatment option become available.
After more than a year of tireless advocacy, a father's campaign has paid off. His 12-year-old son with Duchenne Muscular Dystrophy will finally access a treatment that could help him walk through his entire school career.
Mike Sciotti crawled 11.3 miles on his hands and knees in 1978 to raise money for muscular dystrophy. Nearly five decades later, his college returned to honor the world record that almost nobody remembered.
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