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1881 results for "fragile x syndrome"
Scientists at UCLA have discovered a promising new treatment pathway for Fragile X syndrome, the most common inherited cause of intellectual disability. The breakthrough could help patients of all ages, not just young children.
A promising therapy for fragile X syndrome, a leading genetic cause of autism, is moving to human trials after securing funding from strategic partners. The treatment could also help patients with other rare brain disorders.
Hayden Eastbury turned his love for ABC News into an epic family road trip, visiting 36 bureaus in under two years. His journey has raised national awareness for fragile X syndrome while showing what's possible when families embrace their children's passions.
Researchers at Harvard Medical School have developed a breakthrough gene-editing method that successfully silences the extra chromosome causing Down syndrome in up to 40% of lab cells. While still early stage, this proof-of-concept study opens the door to the first genuine treatment for one of the most common genetic conditions.
Japanese athletes dominated the X Games Chiba competition at Makuhari Messe, bringing home multiple medals in front of roaring hometown crowds. The event marked the second stop of the newly launched X Games League.
Justin Dowell wasn't picked in the X Games draft despite being the reigning champion. He answered his doubters with a near-perfect performance that brought Sacramento's first X Games to life.
Fin Melville Ives from Wanaka, New Zealand, dominated the men's ski superpipe at the X Games, landing tricks he'd never completed in competition before. His victory helped New Zealand achieve its most successful X Games campaign ever.
Twenty-three years ago, Michelle Sie Whitten was told her daughter with Down syndrome would likely die young. Today, Sophia is thriving, and her mom is fighting to give families accurate information about the diagnosis.
Australian snowboarder Scotty James made history at X Games Aspen by landing the first-ever back-to-back backside 1440s, securing his fifth consecutive halfpipe title. The groundbreaking performance ties him with legend Shaun White at eight X Games gold medals.
The U.S. House of Representatives unanimously approved the DeOndra Dixon INCLUDE Project Act, making permanent the first NIH-wide research program dedicated to Down syndrome. The bipartisan legislation honors DeOndra Dixon's legacy and promises to advance discoveries benefiting millions.
Breezy White just became the first person with Down syndrome to graduate from Georgia Highlands College and earn a college degree in Georgia. Her journey from childhood dream to 4.0 GPA student shows what's possible when opportunity meets determination.
A century after his birth, scientist Jérôme Lejeune's breakthrough discovery of Down syndrome's cause now powers a global network helping over 13,000 patients. The foundation bearing his name has become one of the world's leading research centers for genetic intellectual disabilities.
Australian snowboarder Scotty James just made halfpipe history by landing a never-before-seen trick combination to win his fifth consecutive Winter X Games title. The 31-year-old's gravity-defying performance ties him with legend Shaun White at eight total X Games gold medals.
A middle schooler designed a onesie for infants with Down syndrome after watching her brother struggle with regular clothes. Her invention won top prize at Coweta County's Innovation Expo, where 60+ student teams pitched products to solve real problems.
Four astronauts with just four hours of training successfully captured the first X-ray images in space using a portable machine. The breakthrough could make Moon missions safer and bring fast, affordable medical imaging to remote areas on Earth.
Families affected by Hunter syndrome now have hope as the FDA approves Avlayah, the first treatment designed to replace a missing enzyme that causes dangerous sugar buildup in the brain. This breakthrough marks a major victory for patients with this rare genetic disease.
Families across Europe now have access to DAYBU, the first approved treatment for Rett syndrome, a devastating neurological condition that mainly affects young girls. The drug will reach patients in all 27 EU countries plus Iceland, Liechtenstein, and Norway starting in 2026.
Researchers discovered that different mutations in Rett syndrome require different treatments, opening the door to personalized medicine for children with this developmental disorder. Using advanced brain tissue models, they successfully tested mutation-specific therapies that could help thousands of patients.
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 three-year legal battle, X Corp agreed to pay $465,000 for failing to report how it fights child exploitation content on its platform. The ruling marks a major win for online safety regulators holding tech giants accountable.
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