Rare Disease Report

Global Genes Patient Summit 2017

To avoid arginine accumulation, patients with Arginase-1 deficiency must be on an extremely low protein diet.
Stratton said it is the responsibility of the patient community to let  pharma and the FDA know what is important to patients.
monARC Bionetworks provides their mobile apps for free to advocacy groups to help them organize their patient data.
Mark Sorrentino, M.D. of PRA Health Sciences talks about changing clinical trial designs to make them more convenient for patients.
3 billion is a small biotech company that plans to provide kits for people to test their entire exome for rare disease mutations. 
Seizure dogs are trained to recognize changes in scent of their owner’s saliva during a seizure and use that signal to secure the patient.
Accredited researchers can obtain genomic information on a variety of condition, including many rare diseases.
Lipodystrophy is a group of rare diseases characterized by the lack of fat tissue.
This is the 5th anniversary of the event with an estimated 2500 people expected to attend. 
Team Impact is a non-profit organization that brings together collegiate sports teams with children who have rare and chronic conditions.
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