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A total of 1969 posts are filed under Uncategorized
FDA Approves BioMarin’s Enzyme Therapy for PKU
Rare Daily Staff The U.S. Food and Drug Administration approved BioMarin Pharmaceutical’s Palynziq for adult patients with phenylketonuria… Continue Reading
An Ironman Champion with Cystic Fibrosis Discusses her Changing Relationship with Her Disease
Lisa Bentley spent 20 years competing as a professional athlete, although early in her career she kept secret that she suffered from the… Continue Reading
The Case for a Daily Self-Injection to Treat Hemophilia
The treatment of hemophilia has been moving toward longer periods between the dosing of recombinant factors. Now, with the advent of gene… Continue Reading
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

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