is a group of congenital, hereditary neuromuscular disorders that cause muscle weakness, generally nonprogressive, of varying severity where severe forms show up to 66% mortality rates before the age of two. It is considered one of the most severe muscle conditions of childhood. Currently no treatments or disease modifying therapies exist for nemaline myopathy and few potential candidate drugs have been identified.
A Foundation Building Strength is a non–profit organization dedicated to finding treatments for Nemaline Myopathy.
We do this by funding research aimed at understanding protein function and disease progression, prerequisites to regulatory approval such as natural history, and gene expression and editing such as CRISPR, exon skipping, gene therapy, and related approaches.
- October 9 @ 10:15 am - 12:00 pm
Ethan is 2 years old and has a rare muscle disease called Nemaline Myopathy. He is a bright, goofy and sweet little boy......