Explore how collaboration fuels breakthroughs in rare disease research. Join us in uncovering insights that drive change and ...
The FDA’s proposed Rare Disease Evidence Principles review process is a starting point for getting rare disease therapies ...
In recent years, Roche has made a habit of oscillating between first and second place on rare disease patient groups’ annual ...
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 ...
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 ...
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 ...
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 ...
Families of kids with PDCD are urging the FDA to approve DCA, a treatment that could extend their children's lives despite ...
An international team of researchers led by Duke-NUS has identified rare mutations in the SPNS1 gene as the cause of a ...
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