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.
- July 25 @ 7:30 am - July 28 @ 9:00 pm
Join us this summer! Stronger Together: 2019 Scientific and Family Conference, held at the Renaissance Downtown Chicago Hotel on July 25-28, 2019,...
- November 3
AFBS is proud to be selected as an official charity partner of the 2019 TCS New York City Marathon. Five individuals...