Patients & Caregivers
Lysosomal Storage Disorders
Part 3 of Gene Therapy Trial for MPS IIIA (Sanfilippo A) Has Begun
Today, Abeona Therapeutics announced the enrollment and administration of ABO-102 (3 x 1013 vg/kg) of the first 2 patients in Cohort 3.
Children with Sanfilippo Type A Given Gene Therapy Showing Signs of Stabilization
Sanfilippo syndrome type A is a genetic condition in which the build up of heparan sulfate leads to progressive neural degeneration.
New Data Evaluates Origin of Pain in Gaucher Disease
A study published in the Orphanet Journal of Rare Diseases confirmed the role played by peripheral neuropathy in Gaucher pain.
New Data Suggest Clinical Benefit of Idursulfase in Hunter Syndrome
New data indicate that Elaprase (idursulfase) provides a clinical benefit to mucopolysaccharidosis type II (MPS II; Hunter syndrome) patients.
PBS Documentary Gene Doctors Explores Rare Diseases
The Gene Doctors
is available. Film explores the scientific breakthroughs that are transforming rare disease patients
Ultragenyx to Buy Dimension Therapeutics for $151 Million
Ultragenyx to buy small gene therapy company (Dimensions Therapeutics)
NTSAD 2017 Research Grant Recipients
Since 2002, NTSAD has provide 56 research grants for a total of $3.8 million.
Can Pokemon Educate Doctors About Rare Diseases?
An advocacy group is using a card game to educate doctors about ultra-rare diseases.
Avrobio Developing a Gene Therapy for Pompe Disease
It is the third gene therapy the company has in its pipeline to treat a rare lysosomal disease (in addition to Fabry and Gaucher therapies)
New Treatment for MPS VI (Maroteaux-Lamy Syndrome) In Development
Current treatment for MPS VI is ERT [Naglazyme] or hematopoietic stem cell transplant but more treatment options are needed.
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