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 ...

Rare disease forum in Farmington shares latest breakthroughs

FARMINGTON, Conn. (WTNH) — Researchers, biotech executives and policy makers came together Tuesday morning for a rare disease ...
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.

CNBC Cures: Defying Rare Disease documentary preview

An early look at CNBC's new 1-hour documentary, Defying Rare Disease. Defying Rare Disease offers an inside look at 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 Disease Advisory Council within the state Department of Health. The council ...
Fierce Biotech

Rare disease advocates stage 'funeral' at FDA, demand 4 actions of agency and Congress

More than 100 marchers took to Capitol Hill on Wednesday after carrying a coffin to the FDA in a symbolic protest highlighting lives they say could be lost to regulatory delays affecting a group of ...
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 ...

Genome analysis uncovers new cause of rare movement disorder

Despite modern high-throughput sequencing, the genetic cause of most rare movement disorders remains unclear. A research team ...
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 ...

New Alliance Brings Together 1.2 Million Illinois Rare Disease Patients

A chance meeting in a DC hallway turns into a movement SPRINGFIELD, IL, UNITED STATES, March 13, 2026 /EINPresswire.com/ — In the world of rare diseases, finding someone who understands your journey ...
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 ...