Two posters set to be presented at the 65th American ... Patient input and experiences play a crucial role in advancing rare disease research and therapy development, as they help define the ...

1:15 p.m Keynote: Evolution of Rare Disease Research and Medical Care 2:15 p.m. Rare Disease Research Lightening Talks and Poster Session ...

Dominika's one-day-old daughter Emilia is part of the screening programme The entire genetic code of up to 100,000 newborn ...

Results from Phase 3 ENERGIZE-T Study of Mitapivat in Transfusion-dependent Thalassemia will be Presented in Oral Session –– Tebapivat Phase 1 ...

Data from the pivotal Phase 2b ReNeu trial demonstrate that patients with NF1-PN achieved deep responses and improvements in ...

The mother of an eight-year-old boy with a rare disease has spoken of her pride ... Mrs Tilley zip tied posters with the ...

"Mary Poppins" legend Dick Van Dyke proved you are never too old to wade into politics when he proudly endorsed presidential ...

Poster Presentation Details ... The company has a deep pipeline of AI-developed therapeutics spanning oncology, rare disease and neurology, including several in late-stage clinical trials.

SpringWorks to present long-term efficacy and safety data from phase 3 DeFi trial of Ogsiveo in adults with desmoid tumours at CTOS 2024 meeting ...

Screening newborn babies for these rare diseases involves sequencing their ... having seen it advertised on a poster during ...