Patients & Caregivers
Lysosomal Storage Disorders
Kanuma Improves Survival in Infants with LAL Deficiency
New data presented at the NASPGHAN meeting in Las Vegas.
Attention Lysosomal Disease Researchers: WORLDSymposium Now Accepting Late-breaking Abstracts
Attention researchers: you have until December 1st to submit your abstract
Exploring Mortality Rates in Sanfilippo Syndrome Patients
Data indicates some progress in being made in how long these patients survive, the reality is that most are still dying at fairly young ages
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.
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