Lessons from Pitching Venture Capitalists

Stories abound about plucky entrepreneurs…

Ultragenyx Wins FDA Approval of First Therapy for MPS VII
Rare Daily Staff The U.S. Food and Drug Administration approved Ultragenyx Pharmaceutical’s MEPSEVII, the first treatment for children and… Continue Reading
ICER Issues Modified Framework for Assessing Value of Treatments for Ultra-Rare Diseases
Rare Daily Staff The Institute for Clinical and Economic Review, an independent non-profit research institute that analyzes the evidence on… Continue Reading
Trump Taps Former Drug Industry Exec to Head HHS
Daniel S. Levine President Donald Trump said he would nominate Alex Azar, an attorney and former drug industry executive who served in the… Continue Reading
Rare Leader: Sharon King
The Basics Name: Sharon King Title: President Organization: Taylor’s Tale Disease focus: CLN1 disease, a form of infantile Batten disease… Continue Reading
The Process of Being Alive
Daniel S. Levine Claire Wineland laughs in the face of death. In fact, she’s made a bit of a career doing this on her own YouTube channel… Continue Reading
Alnylam Begins Rolling Submission of hATTR Amyloidosis Drug for FDA Approval
Rare Daily Staff RNAi therapeutics developer Alnylam Pharmaceuticals said it has begun submission of a rolling Application to the U.S. Food… Continue Reading
Ultragenyx Completes Dosing of First Cohort in Study of Gene Therapy in OTC Deficiency
Rare Daily Staff Ultragenyx Pharmaceutical said it has completed patient dosing in the first cohort of three patients enrolled in a phase… Continue Reading
Sobi Initiates Late-Stage Study of Kineret to Treat Still’s Disease
Rare Daily Staff Swedish Orphan Biovitrum said that it has randomized the first patient in its late-stage study of Kineret in the treatment… Continue Reading
Naia Rare Diseases Gets Green Light for Clinical Trials of Treatment for Short Bowel Syndrome
Rare Daily Staff Naia Rare Diseases, a biopharmaceutical company developing drugs for rare gastrointestinal diseases, said it has received… Continue Reading
Inozyme Pharma Raises $49 Million to Develop Therapies for Rare Diseases Affecting Soft Tissue and Bone
Rare Daily Staff Inozyme Pharma, a drug developer focusing on rare diseases involving calcification of soft tissues and bone, completed a… Continue Reading
Akcea Initiates Mid-Stage Trial of Therapy in Patients with Rare Hyperlipidemias
Rare Daily Staff Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals focused on developing and commercializing drugs to treat patients… Continue Reading