6 stories tagged with #rare-diseases, in publish-time order across the WeSearch catalog. Tag pages update as new stories ingest.
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Savara: The Rare Disease Funnel Is Becoming A Commercial Asset
Savara is transitioning from a single-asset biotech to a rare disease commercial infrastructure. Click here to read more on SVRA stock.…
Teen's mystery illness solved: "It took 18 years to get an answer"
Lucia Adarve had a long list of symptoms that no one could diagnose. Her mother Lisa was determined to find an answer.…
Ontario girl with Rett Syndrome denied access to promising gene therapy trial, parents say
Parents say the four-year-old was initially selected before eligibility rules changed and later a special access request was cancelled without explanation…
What Rare diseases you think will be cured by 2030 and 2035
STAT+: Biotech execs, academic expert lament impact of FDA turnover on rare disease drug development
How the ouster of key officials at the FDA has created anxiety and uncertainty for the developers of rare disease drugs.…
My son has a terminal disease — why FDA delays are failing families like mine
A mother caring for her son with Duchenne muscular dystrophy urges FDA Commissioner Makary to prioritize faster treatment approvals for rare diseases.…