Tag: MPS

A total of 10 posts are filed under MPS
ArmaGen Presents Data from First Cohort of Phase 1/2a of AGT-182 for Treatment of Hunter Syndrome
ArmaGen, Inc., a privately held biotechnology company focused on developing revolutionary therapies to treat severe neurological disorders,… Continue Reading
Abeona Therapeutics Announces FDA Allowance of Investigational New Drug for Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Abeona Therapeutics, Inc., a biopharmaceutical company focused on developing and delivering products for severe and life-threatening rare… Continue Reading
The Times of India (Bangalore): Court Orders “Treat Rare Disease Patients for Free”
The high court on Monday directed the state run India Gandhi Institute of Child Health (IGICH), Bangalore, to provide free treatment to… Continue Reading
UAB Researcher Answered Questions for Families of Children with Rare Diseases
BIRMINGHAM, Alabama – Few people outside his laboratory at the University of Alabama at Birmingham knew or understood what researcher… Continue Reading
Orphans No More: Renaissance in New Drugs for Rare Diseases
Once famously described as “orphan diseases, too small to be noticed, too small to be funded” in the Hollywood drama Lorenzo’s Oil,… Continue Reading
Charity Tuesday: Courage to Make a Difference
It’s Charity Tuesday and we want to give a shout-out to a new charity called “Courage to Make a Difference” founded by Chris Dutcher.… Continue Reading
A Rare Disease Patient Perspective I would like to use this blog post to explain why the rare disease community is different, and why it… Continue Reading