Alexion announced it will stop developing SBC-103 for the treatment of Sanfilippo type B [mucopolysaccharidosis (MPS) IIIB].
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.
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.
"He has a new lease on life and is looking at things through a different lens, in terms of what is possible"
48-year-old Calgary resident Darren Bidulka is grateful that he no longer has to do the biweekly infusions
A phase 3 trial for MET-overexpressing hepatocellular carcinoma (HCC) failed to reach its endpoint.
Hannah is a young patient battling gigantic axonal neuropathy (GAN).