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93 results for "duchenne muscular dystrophy"
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.
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.
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.
Boys living with Duchenne muscular dystrophy in England now have access to a new treatment that could extend their ability to walk by up to five years. The NHS has approved givinostat, offering fresh hope to families facing this devastating muscle-wasting disease.
Scientists created a breakthrough treatment that successfully restored missing muscle protein in models of Duchenne muscular dystrophy, dramatically improving strength and endurance without the dangerous side effects of current therapies. The new approach could transform treatment for rare genetic disorders and beyond.
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.
A safer gene therapy for Duchenne muscular dystrophy just hit a major milestone, bringing hope to families facing this fatal muscle disease. The treatment could offer a better option than current therapies linked to serious side effects.
A Staten Island father is leading 15 runners through 80 miles of Arizona desert to fund a cure for the disease affecting his 16-year-old son. JAR of Hope has taken teams around the world to raise money for Duchenne muscular dystrophy research.
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.
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.
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.
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.
Boys as young as 2 with Duchenne muscular dystrophy showed remarkable improvements in just 12 weeks when treated early with a safer alternative to steroids. Researchers say starting treatment before symptoms appear could protect muscles before they're lost forever.
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.
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.
A mother who raised $1.3 million to fund a controversial treatment for her son's fatal muscle disease just saw clinical trial results that stunned experts. The experimental drug uses a counterintuitive approach: fixing broken genes by breaking them a little more.
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