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Science & Technology

A total of 112 posts are filed under Science & Technology
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
FDA Helps Fund 21 Early Phase Trials for Rare Disease Treatments
(Source) by Zachary Brennan The US Food and Drug Administration (FDA) on Monday announced it is awarding $23 million in research grants for… Continue Reading
Spark Therapeutics Launches Initiative to Improve Access to Genetic Testing for Inherited Retinal Diseases
Spark Therapeutics (NASDAQ: ONCE) will provide physicians and eligible patients access to genetic testing and counseling for more than 30… 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
Abeona Therapeutics Authorized to Move Forward with Clinical Trial for Sanfilippo Syndrome Treatment
Abeona Therapeutics Inc. (NASDAQ: ABEO), a clinical-stage biopharmaceutical company focused on developing gene therapies for… Continue Reading
Optimizing Market Research Data in Rare Diseases
Story by Claire Ajdukiewicz, a director and Tom Atkinson, group business director at Cello Health Insight; Steve Smith is chief… Continue Reading
Science Magazine: Rare Diseases and Rare Opportunities
On 24 October 2014, after a daylong meeting to plan a rare disease clinical trial, Raymond Wang hopped into a cab and pulled out his… Continue Reading

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