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‘My greatest handicap was the attitude of normal people.’ Alan Marshall’s artful polio memoir, I Can Jump Puddles, turns 70
Polio is in the news, with vaccination under threat and recent outbreaks. This makes Alan Marshall’s classic disability memoir more relevant than ever.
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RARE's GTX-102 Gets FDA's Breakthrough Status for Angelman Syndrome
Ultragenyx Pharmaceuticals RARE announced that the FDA has granted the Breakthrough Therapy designation to its ...
The Manila Times3d
Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome
NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has ...
Ultragenyx Pharmaceutical Inc.: Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome
Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif., June 27 ...
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Angelman syndrome: the disorder of Colin Farrell's son
In a conversation with People magazine in 2024, Colin Farrell opened up for the first time about his son's rare condition: ...