Top Medical News Today

Cerebral Palsy Body Builder Winning More Trophies ... and More Fans
Last month, Steve placed first in his division. Two days later, he was busy training for the next contest (May 2017).
American Health Care Act - Will It Get the Votes?
Today, the American Health Care Act (aka TrumpCare) is being voted in Congress. The bill needs 215 votes to be approved. It is likely that no Democrats support it, meaning that only 21 Republicans can vote against the bill and it can still get approved.
PANDAS: Doctors Divided Over Rare Condition
PANDAS, or pediatric acute-onset neuropsychiatric syndrome, links the sudden onset of OCD or tic-like symptoms to streptococcal-A (GABHS). Could this mysterious affliction be a rare disease or not a disease at all?
Rare Disease Day – A Busy Day for Amicus and Its CEO
To celebrate Rare Disease Day on February 28, 2017, Amicus Therapeutics hosted CabaRare at their Cranbury, N.J. headquarters in which four young people with rare diseases shared their musical talents
Amicus Therapeutics' CabaRare
To help celebrate Rare Disease Day, Amicus Therapeutics brought in 4 young rare disease musicians to share their musical talents with the rare disease community.
Glut 1 DS Phase 2 Study Fails to Meet Primary Endpoint. Ultragenyx Stock Dips 8%
The company said they plan to pursue a Phase 3 study with the drug in Glut 1 DS but that trial will to focus on treating the movement disorders observed in Glut 1 DS patients instead of seizures.
FDA Approves Bavencio (avelumab) for Rare Skin Cancer
The FDA has granted accelerated approval to Bavencio (avelumab) for treating metastatic Merkel cell carcinoma (MCC), a rare skin cancer. This is the first FDA-approved treatment for metastatic (MCC).
4 Years With Eteplirsen – Boys Breathing Better Than Their Counterparts
At the ACMG Annual Clinical Genetics Meeting in Phoenix, AZ, O’Rouke et al presented data showing eteplirsen to slow the progression of pulmonary dysfunction in 12 boys who have been on the drug for 4 years (216 weeks).
Early Detection of CRIM Negative Mutations in Pompe Disease With Newborn Screening
Recent studies examining the benefits of newborn screening for Pompe disease have dramatically changed our perceptions of this rare disease
Beyoncé FaceTimes Teen With Rare Cancer
Thanks to the power of social media, a teen battling a rare form of bone cancer met her idol Beyoncé over FaceTime. Ebony Banks suffers from stage 4 Ewing Sarcoma.
RareDR Resource Guide
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