Top Medical News Today

Dear FDA and Pharma - Your Risk Benefit Ratio is Not My Risk Benefit Ratio
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.
Alexion Shelves Sanfilippo ERT Drug
Alexion announced it will stop developing SBC-103 for the treatment of Sanfilippo type B [mucopolysaccharidosis (MPS) IIIB].
Phase 3 Trial of Agalsidase Beta in Pediatric Patients with Fabry Disease
A Phase 3b trial assessing agalsidase beta in pediatric Fabry disease male patients shows encouraging results.
Clinical Trial Begins in China for Rare Form of Lung Cancer
A Phase II study testing savolitinib in locally advanced or metastatic pulmonary sarcomatoid carcinoma (PSC) has begun in China.
Bench Pressing For Rare Diseases
Garrett Sickels, former Penn State University football player has started a campaign to raise funds for rare disease research while participating in the 2017 NFL Combine.
Newborn Screening for Pompe Disease - A Catalyst For Understanding a Rare Disease
At WORLDSymposium in San Diego, we talked with Priya Kishnani, MD, PhD about the amount of new information gained by studying patients diagnosed at birth with Pompe disease. The data is dramatically changing our understanding of Pompe disease and its management.
Remote Camping with a Disability
"He has a new lease on life and is looking at things through a different lens, in terms of what is possible"
Shire's Rare Disease Products
Hartmann Wellhoefer, MD, head of medical affairs at Shire talks about the company's recent data for Hunter syndrome, Gaucher disease, Fabry disease, and hereditary angioedema (HAE).
The Hidden Costs of Managing a Rare Disease
In an interview with Marielle, a parent of a child with MPSII (Hunter Syndrome) she discusses the other expenses some might not be aware of for caring for a child with a rare disease.
Gene Therapy for Fabry Disease Now a Reality
48-year-old Calgary resident Darren Bidulka is grateful that he no longer has to do the biweekly infusions
RareDR Resource Guide
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