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A total of 170 posts are filed under Research
New Study Being Run for Catalyst Pharma for Patients with Congenital Myasthenic Syndromes (CMS)
Patients may now be eligible to participate in a new research study evaluating an investigational medicine for the treatment of CMS. WHY a… Continue Reading
cTAP Announces Two Research Publications Categorizing and Predicting Disease Progression in Duchenne Muscular Dystrophy
The Collaborative Trajectory Analysis Project (cTAP), a public-private partnership to accelerate data science solutions to critical problems… Continue Reading
CBS Sunday Morning Presents Story on MLD with the The Calliope Joy Foundation
This past Sunday, CBS aired a meaningful segment on late infantile metachromatic leukodystrophy and some of the families who are affected… Continue Reading
Andrew Spear for The Washington Post via Getty
Dr House Goes Digital as IBM’s Watson Diagnoses Rare Diseases
Original Source By Paul Marks Doctor House is going electronic. Medics charged with diagnosing the kind of extremely rare diseases that… Continue Reading
SELLAS™ Life Sciences Announces Positive Interim Phase II Results from its WT1 Immunotherapeutic Anti-cancer Treatment in Multiple Myeloma Patients
SELLAS Life Sciences Group (SELLAS or the Company), a late-stage biopharmaceutical company focused on the development of novel cancer… Continue Reading
University of Arkansas to Lead $1.4M Study on Rare Disease
The University of Arkansas will use a $1.4 million grant to study the origin of Leigh’s Disease, a rare and incurable disease that affects… Continue Reading
Data for BioMarin’s ERT for CLN2 (Batten Disease) is Promising. Very Promising.
Thanks to James Radke PhD and Rare Disease Report for this piece.  The 15th International Conference on Neuronal Ceroid Lipfuscinosis… Continue Reading