Researchers Develop Blood and Urine Test for Autism Spectrum Disorders

Rare Daily Staff Researchers at the University of…

Minoryx Therapeutics Spins Off See-Tx Platform to Create Gain Therapeutics
Rare Daily Staff Minoryx Therapeutics said it has spun off its Site-directed Enzyme Enhancement Therapy (SEE-Tx) platform to form Gain… Continue Reading
Global Genes Rare Disease Stock Index Jumps 8.92% for Week as Market Rebounds
The Global Genes Rare Disease Stock Index rose 8.92 percent as the market rebounded after the recent sell-off. The index outoutperformed… 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
Rare Leader: Carrie Ostrea, Executive Director of NGLY1.Org
The Basics Name: Carrie Ostrea Title: Executive Director Organization: Disease focus: N-glycanase deficiency, or NGLY1 deficiency,… 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