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1587 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.
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.
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.
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.
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.
A groundbreaking treatment could help children with Dravet syndrome not just reduce seizures, but actually improve cognitive development and daily living skills. For the first time, families facing this devastating genetic condition have hope for a disease-modifying therapy.
Media entrepreneur Chude Jideonwo is breaking silence about living with Tourette syndrome after his doctor warned him not to speak publicly about it. His courage is helping change how neurological conditions are viewed across Africa.
Families dealing with Hunter syndrome just gained a powerful new treatment option after the FDA granted accelerated approval to Denali Therapeutics' groundbreaking drug. The decision marks a turning point for rare disease patients after a year of tougher regulatory outcomes.
Scientists at Texas Children's Hospital discovered a promising way to treat Rett syndrome, a rare disorder that affects 1 in 10,000 girls and currently has no cure. The breakthrough could help 65% of patients by boosting levels of a partially working protein in their brains.
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