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 ...
Baylor College of Medicine

NIH awards more than $15 million to fund two rare diseases consortia

Baylor College of Medicine has received new funding from the National Institutes of Health for two consortia in the Rare ...
Napa Valley Register

A look at some key statistics on rare diseases in the U.S.

The global pharmaceutical industry is make huge investments in treatments for rare diseases, which once drew little interest from major drugmakers. A look at some key statistics on rare diseases: ...
Fierce Pharma

Sobi surpasses Roche in rare disease patient groups' reputation rankings

In recent years, Roche has made a habit of oscillating between first and second place on rare disease patient groups’ annual ...
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 ...
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 ...
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 ...
The American Journal of Managed Care

How Health Systems, Pharmacy Teams Tackle Rare Disease Therapy Barriers: David Mitchell, PharmD, MBA

David Mitchell, PharmD, MBA, senior pharmacist manager and assistant clinical professor at UC Davis, addresses how health systems are overcoming financial, operational, and access challenges for ...