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 ...

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

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 ...

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 ...

recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab-lzsg is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation ...

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.

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