Coeur d'Alene Press

Rare disease research shows the power of collaboration

Explore how collaboration fuels breakthroughs in rare disease research. Join us in uncovering insights that drive change and ...
BioSpace

Rare Disease Experts See Futility in FDA’s Proposed Framework

The FDA’s proposed Rare Disease Evidence Principles review process is a starting point for getting rare disease therapies ...
Labiotech.eu

When rare diseases are not so rare after all: A closer look at where and why this happens

Discover how in some communities around the world, the prevalence of rare diseases is higher due to factors like cousin ...
Morning Overview on MSN

AI detects rare diseases from voice

Artificial Intelligence (AI) is significantly impacting the healthcare sector, particularly in diagnosing rare diseases. One ...

Drugmakers Wrestle With FDA Oversight of Rare Disease Treatments

Drug companies that specialize in care for rare diseases are growing frustrated with the FDA, despite agency leadership’s ...
The American Journal of Managed Care

Rare Blood Disease Innovations Address Unmet Needs, According to ASH Data

Rilzabrutinib showed promising results in ITP, achieving significant platelet response and quality-of-life improvements in the LUNA 3 phase 3 study. Fitusiran and ALTUVIIIO studies demonstrated ...
STAT

Rare disease patients caught in Trump crackdown on foreign grant awards

Megan Molteni reports on discoveries from the frontiers of genomic medicine, neuroscience, and reproductive tech. She joined STAT in 2021 after covering health and science at WIRED. You can reach ...
Business Wire

New Study Shows Impact of Ambry Genetics’ Patient for Life™ Program on Rare Disease Diagnosis

Peer-reviewed publication examines the benefits of proactive exome reanalysis on diagnostic outcomes Ambry Genetics’ Patient for Life program is supported by a dedicated team of scientists who ...

Families plead with FDA to approve rare disease treatment

Families of kids with PDCD are urging the FDA to approve DCA, a treatment that could extend their children's lives despite ...

Scientists Solve Medical Mystery Behind Rare Multi-Organ Disease

An international team of researchers led by Duke-NUS has identified rare mutations in the SPNS1 gene as the cause of a previously unrecognized multi-organ disorder. A research team led by Duke-NUS ...