22-YEAR-OLD Kiersten lives with a rare condition called Friedreich's ataxia (FA), which "causes progressive coordination loss" in all of her muscles. Growing up, she was a naturally athletic Varsity ...
Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...
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‘Our kids don’t have time’: NC parents, researchers urge FDA to act quickly on drugs for rare diseases
RALEIGH, N.C. (WNCN) – Parents of children with ultra-rare diseases are encouraging the Food and Drug Administration to act quickly to approve drugs that could treat their conditions and to take into ...
Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for many diseases. By Pam Belluck and Carl Zimmer Gene-editing therapies offer ...
The National Institutes of Health established the Genomics Research to Elucidate the Genetics of Rare Diseases (GREGoR) Consortium in 2021 with the goal of finding molecular diagnoses for individuals ...
Globally, there are more than 300 million people who have one of the 7,000 known rare diseases (Nguengang Wakap et al., 2020). In the United States, any disease affecting fewer than 200,000 people per ...
The Medicines and Healthcare products Regulatory Agency (MHRA) has announced plans to overhaul its approach to therapies for rare diseases. In a position paper, the agency highlighted that rare ...
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