The Genetics of Fibromuscular Dysplasia
Nabila Bouatia-Naji, PhD of INSERM talks about the genetics of fibromuscular dysplasia (FMD) and the studies she is involved with to better understand this rare condition.
SCAD stands for spontaneous coronary artery dissection. It is a rare condition that occurs when a tear forms in one of the blood vessels in the heart.
According to Dr Kim, the number of people with FMD is currently unknown and the prevalence is likely much higher than current estimates.
Project Alive debuted a documentary series to recognize MPS. Each episode will provide a glimpse into the lives of families dealing with Hunter syndrome.
Excellus BlueCross BlueShield is denying to provide the drug on the basis that the boys are not ambulatory and the drug is 'medically unnecessary'.
At 5 milestones, wound healing was observed with most grafts, whereas none of the 4 wounds that weren't treated with the gene therapy showed improvement.
Imagine a young female comes into the ER with abdominal pain and constipation. Would you suspect a rare disease? Probably not.
eSight eyewear is the Star Trek like device that allows legally blind 13-year-old, Ethan LaCroix, the ability to play basketball with the Harlem Globetrotters
uniQure, the first and only company to have a gene therapy approved, will not seek to renew its gene therapy approval in Europe.
Dwight Koeberl, MD at Duke University talks about the latest studies being done to test gene therapy for Pompe disease. The interview took place during Duke University's Pompe disease Patient Meeting held April 8th, 2017.
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