my·op·a·thy:
1. a disease of muscle tissue.
syn·di·cate:
1. a group of individuals or organizations combined
to promote some common interest.
The LGMD1D DNAJB6 Foundation and The Myofibrillar Myopathy (MFM) Group are embarking on a broad mission to include as many autosomal dominant and X linked myopathies and their subset, muscular dystrophies, in a group or syndicate for our common good. Genetic therapies for our distinct genetic problem are a common denominator for approaches like CRISPR editing and RNA blocking techniques. We share the same path forward.
As individual orphan conditions, our numbers are small but collectively we can make a difference. See a partial and growing list of who we are.
Our goal as a syndicate will be political and medical. We encourage all patients, caregivers, friends and interested activists to join. You must be over 18 years old to join or guardian approval. All data is protected.