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9 results for "rare disease treatment"
Jeff Vierstra lost his mother and two sisters to a deadly genetic disease, but an experimental treatment may be preventing him from developing ALS for the first time in his family's history. After three years of spinal infusions targeting his mutated gene, the 41-year-old shows no signs of the disease.
Adults with Fabry disease in the European Union can now receive treatment once a month instead of every two weeks, giving patients and families more time to live beyond managing their condition. The new dosing schedule for Elfabrio could ease the lifelong treatment burden for thousands.
The FDA just approved Denali Therapeutics' new medicine for Hunter syndrome, bringing fresh hope to families affected by this rare genetic disease. The approval comes as a welcome surprise after the agency recently toughened its standards for rare disease treatments.
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.
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.
Children across Europe with a painful swallowing disorder finally have a medicine made just for them. The EU just approved Jorveza oral suspension, ending years of kids taking adult medications not designed for their bodies.
Morocco just launched a Precision Medicine Hub that will help thousands of patients with rare diseases get faster diagnoses and coordinated care. The new national center brings together advanced genetic testing, specialist teams, and patient support under one roof.
A newly approved medication could help people with Prader-Willi syndrome control the constant, overwhelming hunger that defines their daily lives. One mother shares why she's hopeful this treatment will give her son Robert a chance at greater independence and happiness.
Around 20 people in England with a debilitating genetic condition can now access the first treatment that targets the root cause of their disease. The NHS will provide pegzilarginase to patients with arginase-1 deficiency after breakthrough approval.