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Uncategorized

A total of 1966 posts are filed under Uncategorized
FDA Committee Supports Approval of Akcea’s FCS Drug Volanesorsen
Rare Daily Staff A U.S. Food and Drug Administration Advisory Committee voted to recommend that the agency grant marketing approval to… Continue Reading
United Therapeutics to Acquire SteadyMed up to $216 Million
Rare Daily Staff United Therapeutics is strengthening its arsenal by agreeing to acquire orphan drug company SteadyMed for $141 million and… Continue Reading
Casting a Wider Net for Genetic Diseases
While breakthroughs in the ability to diagnose newborns with genetic diseases continues to expand in scope and fall in cost, the extent of… Continue Reading
Why Rare Diseases May Be More Common Than Thought
Rare genetic diseases can be difficult for doctors to recognize, particularly when the symptoms of a disease fall short of dramatic,… Continue Reading
Retrophin $3 million Commitment to Establish Rare Disease Network at Children’s National
Rare Daily Staff Children’s National Health System said it was establishing the Retrophin Rare Disease Network at Children’s National… Continue Reading
RARECast: Understanding Rare Disease Caregiver Needs
Rare disease caregivers are good at advocating for their family members, for research, and for the needs of rare disease patients broadly.… Continue Reading
Health Canada Kills Planned Orphan Drug Framework for Broader Review
Daniel S. Levine Durhane Wong-Rieger has long been working to get regulators in Canada to create an orphan drug designation similar to what… Continue Reading
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