Mucopolysaccharidoses (MPSs) are a group of genetic lysosomal disorders in which persons have low levels of specific enzymes that leads to an abnormal accumulation of complex carbohydrates (mucopolysaccharides or glycosaminoglycans).
KAT6A syndrome is a rare genetic condition as a result of a mutation in the KAT6A gene. That gene produces the KAT6A protein that is instrumental in DNA packaging and the production of other proteins in the cell.
Rare in Common, a podcast hosted by Andra Stratton, regularly posts conversations with leaders in the rare disease community to help them share their stories.
Lynn O’Connor Vos, President and Chief Executive Officer at the Muscular Dystrophy Association, discusses her organization and it’s approach to funding research to accelerate treatments and cures across the full spectrum of neuromuscular diseases.
Lynn O’Connor Vos, President and Chief Executive Officer of the Muscular Dystrophy Association (MDA) provides an overview of her association.
That ease of letting the U.S. take care of the work means that Canada is missing out on early patient access, research funding, and biotech investments.