Rare Disease Report
Patients & Caregivers


Strongbridge Biopharma announced the launch of a free genetic test to diagnose hyperkalemic or hypokalemic periodic paralysis.
The award was presented to Rosbeck by actor Jim O'Heir who regularly participates to the Tuberous Sclerosis Alliance fundraisers

Since 2002, NTSAD has provide 56 research grants for a total of $3.8 million.
This is the 5th anniversary of the event with an estimated 2500 people expected to attend. 
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.
The U.S. Food and Drug Administration has granted Orphan Drug Designation to Revalesio’s investigational drug RNS60 to treat amyotrophic lateral sclerosis.
An advocacy group is using a card game to educate doctors about ultra-rare diseases.
Chaperone drug AT2221 binds to ATB200 to help stabilize the latter ERT until it reaches its target (lysosomes).
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.
 Cure CMD held the International Conference on LAMA2-CMD on March 28-29 this year.
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