How CNBC Cures is bringing rare disease stories to a national audience

"Squawk Box" co-host Becky Quick launched CNBC Cures in January, the network's initiative to raise awareness for rare ...

2026 HNF CMT Summit Convenes FDA Leadership to Accelerate Rare Disease Innovation

Only CMT Event Conducting On-Site Research Brings Regulators, Industry, Researchers, and Patients Together ELLICOTT ...

'CNBC Cures: Defying Rare Disease' premieres Thursday at 7 p.m. ET

Hosted by Becky Quick, CNBC will debut an in-depth, one-hour special, “CNBC Cures: Defying Rare Disease,” spotlighting the ...
Jacobs School of Medicine and Biomedical Sciences - University at Buffalo

Highlighting Advancements in Treatment of Rare Diseases

This year’s Rare Disease Day event Feb. 27 at the Jacobs School of Medicine and Biomedical Sciences focused on updates in research and diagnostics, and advancements in treatment.
CT Insider on MSN

Connecticut rare-disease experts target breakthroughs in life-saving drugs, treatments

Blindness and deafness in some infants and sickle cell are among the conditions cured in recent gene therapy trials.

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

From February 24-26, 2026, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) convened hundreds of patients, caregivers, advocates, researchers, and policymakers from 49 states, the ...
News10NBC

New state bill could bring progress to families dealing with rare diseases

ALBANY, N.Y. — A bill in the state Legislature aims to raise awareness of rare diseases. The bill would establish a Rare ...
MM&M

Pediatric rare diseases are often underdiagnosed. GeneDx wants to change that.

GeneDx, which specializes in diagnostics for rare and ultra-rare genetic disorders, is helping to get kids the diagnostic attention they need as part of Diagnosis is Power. Currently, one in 10 ...
STAT

Parents of children with rare diseases ask: How long until our CRISPR miracle?

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 ...
STAT

‘Where’s our CRISPR miracle?’

Torie Bosch is the First Opinion editor at STAT. Celena Lozano’s son Benny, who turned 5 in November, loves trains, trucks — anything that goes. He also has a rare disease, PURA syndrome. Earlier this ...
Morningstar

National Organization for Rare Disorders Adds Seven Rare Disease Centers of Excellence Nationwide

NORD's growing network, now 46 institutions strong, represents a coordinated nationwide effort to strengthen rare disease care and research collaboration With these new members, the network now ...