Why Rare Disease Patients Should Worry About the Shifting Discussion from Value to Scarcity

The high cost of rare disease therapies is often…

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
Amryt and Aegerion Expand Agreement for HoFH Drug in Russia, CIS, and Non-E.U. Balkan States
Rare Daily Staff Amryt said that it expanded its exclusive license agreement with Aegerion Pharmaceuticals for the rare cholesterol disorder… Continue Reading
Rare Leader: Kelly Ranallo, Founder and President, Turner Syndrome Global Alliance
The Basics Name: Kelly Ranallo Title: Founder and President Organization:  Turner Syndrome Global Alliance Disease focus:  Turner syndrome… Continue Reading
Alexion Reports Positive Top-Line Results from Late-Stage Study of Soliris Successor
Rare Daily Staff Alexion Pharmaceuticals said that the pivotal late-stage study of ALXN1210, the company’s experimental long-acting… Continue Reading
Takeda Agrees to Acquire Shire for $62.4 Billion
Rare Daily Staff Takeda Pharmaceutical and Shire said that they have reached agreement on the terms for Takeda to acquire the rare disease… Continue Reading
ICER Blasts Pricing of Vertex CF Drugs, Vertex Blasts Back Calling Process a “Sham”
Rare Daily Staff The Institute for Clinical and Economic Review said its analysis of the comparative clinical effectiveness and value of… Continue Reading

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