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11 results for "muscular dystrophy"
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.
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.
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.
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 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.
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.
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 South Korean teen with muscular dystrophy can now lift his arms with ease thanks to a revolutionary shirt that weighs less than two pounds. The wearable exoskeleton costs thousands less than traditional models and could restore independence to millions.
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 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.