Delay is a death sentence. Why rare disease families don’t trust Trump’s FDA nominee

We call it the “rare disease community,” but there is nothing rare about the number of Americans whose lives are touched by ...
Medscape

Interorgan Cross Talk Insights and Cross-Border Registries Advance Care for Rare Pediatric Lung Diseases

Living with a rare lung condition can be especially challenging, but research into interogran cross talk and organoid models, and cross-border registry are improving outcomes.
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 ...

Rare zoonotic disease found in East Glacier beaver

A beaver in Glacier National Park tested positive for a rare but serious zoonotic disease known as tularemia. While most commonly associated with hares, rabbits and rodents, the bacterial infection ...
Labroots

Diagnostic Gene Hotspot Found in a Rare Childhood Muscle Disease

Clinical analysis of 49 patients with congenital nemaline myopathies (NMs), an ultra-rare class of muscle diseases that cause muscle weakness and respirato | Genetics ...
STAT

I’m a rare disease mom, and I finally have new hope for my son’s future

Stecker is a rare disease mother and advocate and served as assistant secretary of public affairs and deputy chief of staff at the Department of Health and Human Services. Every parent dreams of a ...
BIOENGINEER

Six Experts Map the Future of Rare Metabolic Disease Research

Rare diseases are defined individually by their scarcity, but collectively they represent one of the largest burdens of unmet medical need in modern medicine. An estimated 300 million people worldwide ...

Glacier Park: Rare disease detected in beaver

Glacier National Park officials announced last week a beaver discovered on the east side had tested positive for a rare but serious bacterial disease. Tularemia occurs naturally and can affect both ...
Forbes

New $25 Million Accelerator Bets On Better Model For Rare Disease

This voice experience is generated by AI. Learn more. This voice experience is generated by AI. Learn more. A new Pittsburgh-based initiative, Rare Ventures, has launched with a $25 million commitment ...
AOL

Michigan confirms human case of rare mosquito-borne disease

Aug 19 (Reuters) - The Michigan health department on Wednesday confirmed a locally acquired case of eastern equine encephalitis, a rare mosquito-borne disease. The patient, a resident of Roscommon ...
Health and Me on MSN

Google DeepMind’s AlphaGenome Atlas maps 9 billion mutations in human genome; to reveal genetics, rare disease clues

AlphaGenome Atlas dataset is about 1 petabyte in size, making it more than 30 times larger than the previous AlphaFold ...
Bozeman Daily Chronicle

Glacier Park: Rare disease detected in beavers

Glacier National Park officials announced last week a beaver discovered on the east side had tested positive for a rare but serious bacterial disease.