Care information
Limb-Girdle Muscular Dystrophy (LGMD) Family Guide
Developed by TREAT-NMD and Sarepta Therapeutics, this plain-language guide provides practical information for people living with LGMD, their families, caregivers, and healthcare professionals. Created with input from international clinical experts and patient advocates, it covers general LGMD information and several common subtypes, with plans to expand to additional subtypes and languages over time.
Limb-Girdle Muscular Dystrophy (LGMD) Family Guide
Congenital Muscular Dystrophy Consensus Guidelines
The CMD Consensus Guidelines represent the efforts of an international CMD clinical consortium led by Dr. Ching Wang, Dr. Thomas Sejersen and Dr. Anne Rutkowski and was made possible through the generous support of TREAT-NMD, AFM, Telethon and Cure CMD.
The International Standard of Care Committee for Congenital Muscular Dystrophy was established to identify current care issues, review literature for evidence-based practice, and achieve consensus on care recommendations in 7 areas: diagnosis, neurology, pulmonology, orthopaedics/rehabilitation, gastroenterology/nutrition/speech/oral care, cardiology, and palliative care.
Congenital Muscular Dystrophy Consensus Guidelines
A Family Guide has been produced (based on the full academic article) and is a foundation of information for families so that individuals and families can use it in collaboration with their care providers to discuss their own specific needs.
Management of CMD: A guide for families
Congenital Muscle Disease International Registry (CMDIR)
The Congenital Muscle Disease International Registry, including Congenital Muscular Dystrophy, Congenital Myopathy, and Congenital Myasthenic Syndrome, was created to identify the global congenital muscle disease population for the purpose of raising awareness, standards of care, clinical trials and in the future a treatment or cure. The CMDIR registers through the limb girdle spectrum for all three disease groups.
If you have Congenital Muscular Dystrophy (CMD) not related to an FKRP mutation, or if the underlying gene mutation is not yet known, please register on the CMDIR. It is not necessary to register on both.
LGMD subtyping tool
There is a free online tool to help guide doctors toward the most probable diagnosis for patients who fall into the LGMD category, called the Automated LGMD Diagnostic Assistant (ALDA). This tool predicts the most likely type(s) of LGMD a patient may have based on clinical presentation and laboratory findings.
If you are a patient interested in using the LGMD diagnostic tool, please contact either your doctor or the LGMD2I Research Fund to help guide you through the process.
Click here to use the LGMD Diagnostic Tool
McColl Lockwood Laboratory for Muscular Dystrophy Research
The McColl Lockwood Laboratory for Muscular Dystrophy Research was set-up to develop experimental therapies for the treatment of the muscular dystrophies and to facilitate the translation of experimental therapies to clinical trials for improving the quality of life for patients with the disease.