WNEM on MSN

Creed’s comeback: A new era for fighting a rare childhood disease

Fourteen-year-old Creed Kolasa is funny, competitive, and, like most teens, just wants to have fun. And Creed is doing it all ...
CNBC

Every Cure’s drug repurposing strategy isn't what you think, and it could change rare disease treatment

Every Cure is using AI to seek new uses for the roughly 4,000 existing drugs, and cutting traditional preliminary research timelines from as much as 100 days to as little as 17 hours. Treatment for a ...

Rapid genomic testing helps 1,100 families worldwide target treatment for rare childhood disease

An international partnership designed to improve equality in access to genomic medicine for a rare disease has now provided ...
CNBC

A lost voice, a rare disease and the family conversations that followed

Our inbox has been flooded with emails from viewers and readers who told us how their lives have been affected by rare diseases. Here are some of the stories we received. Living with a rare disease ...
MarketWatch

EveryLife Foundation for Rare Diseases Emboldens Advocates and Urges Congress to Prioritize Patients During 15th Annual Rare Disease Week on Capitol Hill

WASHINGTON, March 10, 2026 /PRNewswire/ -- From February 24-26, 2026, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) convened hundreds of patients, caregivers, advocates, ...
FierceBiotech

Small pools, big ideas: How the rare disease community is reimagining clinical trials

The traditional gold standard of clinical trials with a randomized, placebo-controlled design involving hundreds of patients has inherently been unfeasible for the rare disease community. So ...
Morningstar

The EveryLife Foundation for Rare Diseases Marks 15th Annual Rare Disease Week on Capitol Hill

WASHINGTON, Feb. 18, 2026 /PRNewswire/ -- For the 15th consecutive year, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) will welcome advocates from 49 states, the District of ...
Seeking Alpha

Chiesi Global Rare Diseases and Protalix BioTherapeutics Announce European Commission Approval of Additional Dosing Regimen of Every Four Weeks for Elfabrio® (pegunigalsidase ...

This press release is intended for US audiences for transparency relative to global news for the Fabry community. This dosing regimen for Elfabrio is not approved in the US. In the US, the ...