Prothena Shares Tumble as Company Discontinues AL Amyloidosis Candidate

Rare Daily Staff Prothena said it is discontinuing development of NEOD001, an investigational antibody that was being evaluated for the treatment of AL amyloidosis follow the failure of the experimental therapy meeting its primary or secondary endpoints in a mid-stage trial. Shares of the company’s…Continue Reading

Seeking Answers for Undiagnosed Patients

For patients with a rare disease, the diagnostic odyssey can be a long journey fraught with many wrong answers. The Undiagnosed Diseases Network is a research study funded by the National Institutes of Health to bring together clinical researchers from across the country armed with…Continue Reading

Dicerna to Pay Alnylam $25 Million as Part of Litigation Settlement

Rare Daily Staff Alnylam Pharmaceuticals and Dicerna Pharmaceuticals said they have resolved all litigation relating to lawsuits filed against each other relating to claims and counterclaims of misappropriation of trade secrets over their RNAi technologies. Under the terms of the agreement, Dicerna will pay $25 million comprised…Continue Reading

FDA Clears Ultragenyx to Begin Clinical Trials of Gene Therapy for Glycogen Storage Disease Type Ia

Rare Daily Staff The U.S. Food and Drug Administration gave Ultragenyx Pharmaceutical clearance to begin clinical trials of DTX401, an adeno-associated virus vector-based gene therapy for the treatment of glycogen storage disease type Ia, a genetic enzyme deficiency that result in the inability to regulate…Continue Reading

The Global Genes Rare Disease Stock Index Drops 0.66 percent on Week

Markets turned down on Friday as investor shifted attention away from corporate earnings to concerns about inflation. For the week, the Global Genes Rare Disease Stock Index fell 0.66 percent. The GGRDSI continues to significantly outperform the other indices tracked by Rare Daily with a 20.85…Continue Reading

Rare Leader: Philip Robert, Founder, CEO, and Chief Encouragement Officer of The Burning Limb Foundation

The Basics Name: Philip Robert Title: Founder, CEO, and Chief Encouragement Officer Organization: The Burning Limb Foundation Disease focus:  Complex Regional Pain Syndrome. CRPS is a rare disorder of the sympathetic nervous system that is characterized by chronic, severe pain that is out of proportion…Continue Reading

FDA Commissioner Seeks 13 Percent Funding Increase for fiscal 2019

Rare Daily Staff U.S. Food and Drug Administration Commissioner Scott Gottlieb went before lawmakers earlier this week to make the case for the Trump administration’s proposed $5.8 billion budget for the agency in fiscal 2019. The Trump administration’s budget request includes represents a $663 million…Continue Reading

FDA Grants Modus Therapeutics’ Sevuparin Rare Pediatric Disease Designation for Treatment of Sickle Cell Disease

Rare Daily Staff The U.S. Food and Drug Administration granted Modus Therapeutics’ sevuparin Rare Pediatric Disease designation for the treatment of sickle cell disease, a rare genetic disease that impairs the ability of red blood cells to transport oxygen. The Rare Pediatric Disease Priority Review…Continue Reading

NEJM Publishes Promising Bluebird Bio Gene Therapy Data in Beta Thalassemia

Rare Daily Staff Interim data published in the New England Journal of Medicine from two separate studies of Bluebird Bio’s LentiGlobin, a gene therapy to treat transfusion-dependent beta thalassemia, showed the majority of patients have not required transfusions for two years or longer following treatment. Transfusion-dependent beta…Continue Reading

Shire Rejects Takeda’s $61.9 Billion Bid as Allergan Says It May Jump into the Fray

Rare Daily Staff Rare disease drugmaker Shire said it has rejected a $61.9 billion takeover bid from from Japanese drug giant Takeda as Allegan says it is considering making a competing bid. Takeda latest bid is the third it has made for Shire. As with…Continue Reading