Nemaline myopathy is a neuromuscular disorder which affects the voluntary muscle and is usually non-progressive. The severest form appears at birth, and death can occur in the first few days, weeks or months of life due to respiratory failure. The most common form of the disease is not fatal. Nemaline means thread-like, and researchers chose this name because of the presence in affected muscle cells of threadlike or rod-shaped material. The significance of these rod-like bodies is still not clear. Symptoms are weakness in the skeletal muscles (leg, arm and trunk). Most people with NM have long, narrow faces with high-arched palates, slender bodies and curvature of the spine are common. The jaw, tongue and throat muscles may also be affected, leading to speech and swallowing difficulties. A study in Finland estimated that only 1 in 50,000 live births have nemaline myopathy, making it one of the rarest muscle disorders. The main research labs are in Finland, Australia, USA, with smaller teams in Europe.

Convention delegates from Canada, Europe, Israel, S. Africa and USA, will socialize in a relaxing hotel, listen to scientists and support staff, learn from each other and swap life stories and useful anecdotes.

NM is a very rare (1:50,000) neuromuscular disorder which affects the muscles and respiratory system. The severest form usually appears at birth and death can occur in the first few weeks or months of life due to respiratory failure.