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7104 results for "bone disease"
A new medicine called Pasatru just won FDA approval for fibrodysplasia ossificans progressiva, a devastating disease that turns muscle into bone. The breakthrough could help patients maintain mobility and live longer after three decades of research.
Scientists at McGill University discovered a molecular switch that could revolutionize treatment for a rare bone disorder affecting communities across Canada. The breakthrough reveals how the body activates an alternative energy-burning pathway, opening doors to new therapies.
Adults with a rare disease that turns muscles into bone finally have an FDA-approved treatment that cuts new bone formation by 90%. The drug offers hope to about 900 people worldwide who face progressive loss of mobility from fibrodysplasia ossificans progressiva.
When 5-year-old Emma was diagnosed with a rare bone disease, her 2-year-old sister Lucy became her perfect genetic match and lifesaving donor. The toddler's brave bone marrow transplant in February has given Emma a complete recovery and created an unbreakable bond between the sisters.
Neena Nizar became the first patient in a clinical trial for Jansen's disease, a bone disorder so rare only 30 people worldwide have it. Her determination opened the door to treatment that never existed before.
Uganda is raising $4 million to build its first bone marrow transplant center, offering hope to 20,000 children born annually with sickle cell disease. The facility could cure patients who currently face $300,000-500,000 treatment costs abroad.
A 24-year-old woman with vanishing bone disease now walks without crutches after Israeli surgeons used 3D-printed implants to lengthen her leg by eight centimeters. The groundbreaking procedure replaced her missing hip and thigh bone, restoring mobility she never thought possible.
Researchers discovered how a molecule called glycerol activates brown fat to burn energy and strengthen bones, offering hope for patients with rare bone disorders. The breakthrough could lead to new treatments that help restore healthy bone formation.
Researchers discovered a receptor that could rebuild weakened bones in people with osteoporosis, not just slow the disease. Early tests show the treatment dramatically strengthened bones in mice, offering hope for millions suffering painful fractures.
Researchers at McGill University discovered a molecular switch in brown fat that burns calories and could lead to new treatments for bone disease. The breakthrough reveals how glycerol activates a hidden energy system that also affects bone strength.
For the first time ever, children and adults with Alexander disease have a treatment that attacks the root cause of their condition, not just the symptoms. The FDA's approval of zilganersen gives hope to families facing a disease that has always been fatal.
A groundbreaking treatment for Huntington's disease just cleared a major hurdle, bringing hope to 240,000 Americans affected by this devastating condition. The experimental therapy could become the first disease-modifying treatment for a disorder that has had none.
After seven surgeries forced her to drop out of school, 23-year-old Radhika JA from Coimbatore turned old newspapers into art. Her handcrafted dolls now ship worldwide, transforming childhood isolation into entrepreneurial success.
A doctor who survived a rare disease now uses artificial intelligence to find unexpected treatments that help patients like Kaila Mabus, who was told she might not survive. His AI system tests 75 million possible drug-disease combinations in minutes.
A groundbreaking treatment for myotonic dystrophy type 1 reduced toxic proteins by 40% and improved muscle function in patients who've never had disease-modifying options. The New England Journal of Medicine just published results that could lead to the first approved therapy for this progressive, often fatal disease.
Researchers discovered how to disguise healthy mitochondria so they can slip into diseased cells undetected, extending the lives of mice with a fatal genetic disorder. The breakthrough could one day help children born with rare mitochondrial diseases.
A new treatment for a rare kidney disease just passed a major test, bringing hope to 330,000 patients in the U.S. and Europe. Vertex's drug successfully reduced a key disease marker by half in late-stage trials.
University of Queensland researchers have developed a breakthrough drug that could transform Motor Neurone Disease from a terminal diagnosis into a manageable chronic condition within five years. The discovery unlocks a previously untargetable immune receptor that plays a key role in neurodegenerative diseases.
Pearl Jam's Eddie Vedder and Canadian director Matt Finlin created a documentary following the race to cure epidermolysis bullosa, a devastating skin disease affecting children worldwide. The Netflix film captures breakthrough research that could unlock treatments for thousands of rare diseases.
For the first time, a gene therapy has shown dramatic results in slowing Huntington's disease, offering hope to 8,000 UK families who've had no treatment options until now. Patients receiving the higher dose experienced 75% less disease progression over three years.
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