Why a Sports Physical May Trigger a Doctor to Think About a Rare Diagnosis

Marfan syndrome is a genetic disorder that affects…

UPenn’s Orphan Disease Center Takes a Rare Approach
    Rare disease centers at academic institutions are usually places that focus on early-stage research of faculty members, but the Orphan… Continue Reading
FDA Grants Priority Review for Genentech’s Rituxan for Rare Autoimmune Condition
Rare Daily Staff The U.S. Food and Drug Administration granted Genentech priority review status for its application to expand approved uses… Continue Reading
PCORI Awards $50K to International WAGR Syndrome Association for Conference on Wilms Tumor
Rare Daily Staff The Eugene Washington PCORI Engagement Awards program, an initiative of the Patient Centered Outcomes Research Institute… Continue Reading
Tremeau Closes $5.2 Million Equity Round to Repurpose Vioxx for Rare Diseases
Rare Daily Staff Tremeau Pharmaceuticals said that it raised a total of $5.2 million in its first equity round to advance its pipeline of… Continue Reading
Oxford BioMedica Enters Gene Therapy Collaboration with Bioverativ Worth up to $105M
Rare Daily Staff Oxford BioMedica entered into a collaboration and license agreement worth up to $105 million with Bioverativ for the… Continue Reading
ProQR Enters $7.5 million Partnership with Foundation Fighting Blindness to Develop Usher Syndrome Therapy
Rare Daily Staff The Foundation Fighting Blindness and ProQR have entered into a partnership that will provide the company up to $7.5… Continue Reading
FDA Accepts Amicus Fabry Drug for Priority Review
Rare Daily Staff The U.S. Food and Drug Administration has accepted the New Drug Application and granted priority review status for Amicus… Continue Reading
NICE Draft Guidance Says BioMarin’s ERT for Batten Disease Not Cost Effective
Rare Daily Staff The United Kingdom’s National Institute for Health and Care Excellence issued a preliminary recommendation not to… Continue Reading