Building awareness and communities to support and find treatments for Rare Diseases
Building awareness and communities to support and find treatments for Rare Diseases
Seeking treatment for a rare disease can be complicated by the poor understanding clinicians may have of a given condition and variations in the way they diagnose, treat, and monitor a specific disease. One way to ensure patients receive the best care is through the…Continue Reading
The Basics Name: Skye Miu Steppe Title: Founder and Chairman Organization: FD Warriors Disease focus: Fibrous dysplasia (FD) is a rare bone disease that can affect any bone in the body. The severity of the disease covers a wide spectrum. It can affect a single…Continue Reading
Rare Daily Staff A federal bill intended to address what its authors call the “ever-widening funding gap” between basic research and clinical development would authorize a pilot program that allows the U.S. Treasury Department to establish a privately owned and operated investment fund for rare…Continue Reading
When the European Medicines Agency approved Myozyme in 2006, Genzyme’s enzyme replacement therapy for the lysosomal storage disorder Pompe’s disease, it involved an 18-month randomized, placebo-controlled trial study of 90 patients with the late-onset form of the condition. But the study also included a separate…Continue Reading
RARE Daily is starting a new feature called Voices from the Rare Community and we’d like you to be a part of this. From time to time we’ll be posing questions and we’d like you to send us a video response. For our first…Continue Reading
Rare Daily Staff Amicus Therapeutics agreed to acquire Celenex in a deal that provides it worldwide development and commercial rights for ten gene therapy programs for $100 million upfront and up to $277 million in milestone payments. Through the acquisition, Amicus gains rights to ten programs…Continue Reading
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