February 19, 2016
FIND TREATMENT FOR NM
For its fourth annual fundraiser benefitting A Foundation Building Strength (AFBS), LoveLiv will once again have a Soul Cycle ride! AFBS remains the only non-profit worldwide dedicated to finding treatment for Nemaline Myopathy, a rare muscle disease that Liv has. People with NM, including Liv, generally cannot eat, breathe and walk on their own. Life expectancy is greatly reduced due to respiratory complications. Although Liv has made great strides, she just turned 4 years old and still cannot walk or crawl, and relies on a feeding tube for some of her nutrition. Like so many with NM, her speech is affected by severe muscle weakness in her mouth.
With your help, we can find treatment to better Liv's life and those with NM. AFBS is seeking to fund a gene therapy study for Nebulin based Nemaline Myopathy, targeting the same mutation Liv has. If this study is successful, Liv could have treatment in a few years. This is one of the most exciting proposals we have received and we need your help!
A FOUNDATION BUILDING STRENGTH IS A NON–PROFIT ORGANIZATION DEDICATED TO FINDING TREATMENTS FOR NEMALINE MYOPATHY
Nemaline myopathy (NM) 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.
“Myopathy” means “muscle disease”, and a biopsy of muscle from a person with nemaline myopathy shows abnormal thread–like rods, called nemaline bodies, in the muscle cells. People with nemaline myopathy (or NM) usually experience delayed motor development and weakness in the arm, leg, trunk, throat, and face muscles.
The disorder is often clinically categorized into several groups, including mild (typical), intermediate, severe, and adult–onset; however, these distinctions are somewhat ambiguous, as the categories frequently overlap. Respiratory problems are a primary concern for people with all forms of NM, and though in some severe cases they may threaten life expectancy, aggressive and proactive care allows most individuals to survive and lead active lives.
Our mission is simple: To find treatment for Nemaline Myopathy.