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Uncategorized

A total of 1973 posts are filed under Uncategorized
Ferris Bueller and the Cost of Pivotal Clinical Trials
It turns out asking what a pivotal phase 3 clinical trial costs is a little like asking, “What does a car cost?” The answer will depend… Continue Reading
Rare Leader: David McIntyre, President, Bethanys Hope Foundation
The Basics Name:  David McIntyre Title: President Organization:  Bethanys Hope Foundation Disease focus:  Metachromatic Leukodystrophy… Continue Reading
Claire Wineland, Inspirational Rare Disease Advocate, Dies at 21
Claire Wineland, who rose to prominence as a teenager through her YouTube videos filled with charm and wisdom as she discussed her life with… Continue Reading
FDA Approves Kyowa Kirin’s Poteligeo for Rare Forms of NHL
Rare Daily Staff The U.S. Food and Drug Administration today approved Kyowa Kirin’s Poteligeo for the treatment of adult patients with… Continue Reading
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
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