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A total of 327 posts are filed under Innovation
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
FDA Approves Lundbeck’s IV Carbamazepine For Certain Seizures
By James Radke, PhD at Rare Disease Report The FDA has approved Carnexiv (carbamazepine) injection as a short-term replacement therapy for… Continue Reading
Child Neurology Foundation: New Webinar Friday, October 7, 2016 @ 1pm (EST)
Friday, October 7, 2016 @ 1pm (EST)  Transitioning from the pediatric to the adult health care system should be expected for all… 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
New App for Cystic Fibrosis Patients Helps to Manage Symptoms
People with cystic fibrosis (CF) need help to ensure they are getting correct nutrition and the right amount of enzymes. They also need… Continue Reading
FDA Approves Vertex’s Orkambi for Cystic Fibrosis Children 6-11 Years Old
by Andrew Black for Rare Disease Report The FDA has approved Vertex Pharmaceuticals’ drug Orkambi (lumacaftor/ivacaftor) for use in… Continue Reading