Gaucher Disease
Gaucher Disease and Gene Therapy: Approaches and Research Progress
According to a story from the National Gaucher Foundation, the use of gene therapy in order to treat Gaucher disease has long been floated in
Rare Patient News. Well Done.
Review of Rare Movement Disorders in India and the United States
- March 30, 2020
New Phenylketonuria Treatment Granted Orphan Drug Designation
Experimental Treatment for Cryopyrin-Associated Periodic Syndrome (CAPS) Shows Potential in Early Trial
Immunocompromised During COVID-19? Not Much Has Changed in the Lives of Many Rare Patients
LATEST PATIENT WORTHY ARTICLES
Rare Disease
Long Term Data Looks Good for Spinal Muscular Atrophy Gene Therapy Zolgensma
According to a story from Benzinga, the Novartis Company AveXis recently announced the results of long term studies testing the impact of Zolgensma, a gene
Rare Disease
Why Was a Drug Being Tested for Coronavirus/COVID-19 Given Orphan Drug Designation?
According to a story from The Intercept, Gilead Sciences’ antiviral drug remdesivir, which is being tested as a treatment for coronavirus/COVID-19, was just given Orphan
Rare Disease
Chiesi Launches Global Rare Disease Division Headed in Boston
As originally reported in European Pharmaceutical Manufacturer Magazine, Chiesi, an Italian pharmaceutical company, has announced they will be launching a new division for rare disease
Primary Immunodeficiencies
What if the World Always Thought of the Immunocompromised as They are Right Now?
If the world were as careful in everyday life Perhaps I’d be sick less, I’d feel much less strife It’s the small precautions, like washing
Inflammatory Bowel Disease
Organs-On- A-Chip: A Novel Approach to Studying Inflammatory Bowel Disease
Biological engineers at MIT are using a new technique to study various organs and their interaction with our immune system. According to a recent article
