Patients & Caregivers
Free Genetic Testing for Periodic Paralysis Available
Strongbridge Biopharma announced the launch of a free genetic test to diagnose hyperkalemic or hypokalemic periodic paralysis.
RARE Champion of Hope Collaboration in Advocacy
The award was presented to Rosbeck by actor Jim O'Heir who regularly participates to the Tuberous Sclerosis Alliance fundraisers
NTSAD 2017 Research Grant Recipients
Since 2002, NTSAD has provide 56 research grants for a total of $3.8 million.
Epilepsy Awareness Day at Disneyland
This is the 5th anniversary of the event with an estimated 2500 people expected to attend.
ALS Expert Mary Sedarous is Hopeful for FDA-Approved Radicava
Mary Sedarous discusses Radicava, importance of data that will be available now that patients have access to the drug, and why the approval is already a success.
FDA Pushes ALS Drug Through the Pipeline with Orphan Drug Designation
The U.S. Food and Drug Administration has granted Orphan Drug Designation to Revalesio’s investigational drug RNS60 to treat amyotrophic lateral sclerosis.
Can Pokemon Educate Doctors About Rare Diseases?
An advocacy group is using a card game to educate doctors about ultra-rare diseases.
Pompe Combination Therapy Gets Orphan Designation
Chaperone drug AT2221 binds to ATB200 to help stabilize the latter ERT until it reaches its target (lysosomes).
A Grand Effort: JAR of Hope Founder Raises Duchenne Awareness from the Grand Canyon
Raffone, the founder and CEO of JAR of Hope, is training for the 2017 Grand to Grand Ultra, a 170-mile race to raise awareness for Duchenne muscular dystrophy.
Scientists Gathered for Conference on LAMA2 Congenital Muscular Dystrophy
Cure CMD held the International Conference on LAMA2-CMD on March 28-29 this year.
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