Raredr

Sharing News from NORD Member Organizations

Guest post by NORD
Published Online: Tuesday, Aug 25, 2015
NORD is pleased to be collaborating with Rare Disease Communications, and we are particularly happy about the opportunity this Voice of the Community column will provide to share information from our member organizations. NORD membership is comprised of a diverse array of rare disease patient organizations that have met standards for excellence established by the NORD board of directors.

Currently, we have more than 230 members who together represent hundreds of diseases. Our members and their medical advisors partner with NORD to provide advocacy, awareness and education on behalf of the entire rare disease community. In addition, 84% of NORD’s members are actively involved in research and 79% are actively funding research.

We look forward to providing up-to-the-minute information about important advances, events and resources in this Voice of the Community column, and we would welcome questions or suggestions submitted through the Rare Disease Report editors at any time.  

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Dave Esposito, father of a child with Sanfilippo syndrome, wants the FDA and the pharmaceutical industry to know that their definition of a good outcome measure is different than a parent's definition.
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A Phase 3b trial assessing agalsidase beta in pediatric Fabry disease male patients shows encouraging results.
A Phase II study testing savolitinib in locally advanced or metastatic pulmonary sarcomatoid carcinoma (PSC) has begun in China.
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