Latest Rare diseases Stories
The ASF joins the international Angelman syndrome community in celebrating International Angelman Day on Sunday, Feb.
Three Oral Presentations Provide New Analyses of the Efficacy and Safety of Defibrotide in Patients with VOD, Including Post-Hoc Sub-Group Analyses in Children, Adults, and Allograft and Autograft
Novel ZFN Genome Editing Approach Results in Sustained Levels of Enzymes Associated with Hunter, Hurler and Gaucher Syndromes RICHMOND, Calif., Feb.
LEXINGTON, Mass., Feb.
A woman with WHIM syndrome shocked doctors when her symptoms miraculously disappeared in response to gene shattering.
Researchers at Nationwide Children's Hospital have developed a way to measure upper extremity movement in patients with muscular dystrophy using interactive video game technology. Their hope is to expand inclusion criteria for clinical trials to incorporate patients using wheelchairs.
Nation’s premier Internet resource for clinicians, advocates and patients unveils Rare Disease Report™, a new print publication, during World Symposium in Orlando, expanding RDC’s influence
LYNBROOK, N.Y., Feb. 2, 2015 /PRNewswire/ -- BioSpecifics Technologies Corp.