Cure CMD (Congenital Muscular Dystrophy) is pleased to announce the recipient of a grant co-funded with Muscular Dystrophy UK: Dr. Giséle Bonne at the INSERM-Myology Institute in Paris. Her work will look at genetic changes that alter the severity of LMNA-CMD. Together with Cure CMD, Muscular Dystrophy UK is collaborating to support a program of world-class research to find effective treatments and cures for LMNA CMD.
Dr. Gustavo Dziewczapolski, Cure CMD Scientific Director, explains, “Dr Bonne has been instrumental over the years in helping advance CMD research and particularly LMNA-CMD. Dr. Bonne’s promising project will study factors, called gene modifiers, which modulate the severity of the LMNA-related muscular dystrophies. The results will potentially help to better define prognosis, provide adapted care to patients, and uncover new therapeutic targets. They could also be extremely useful when designing clinical trials, as it may allow for more homogeneous study cohorts to be selected, thus helping reduce variability of trial outcomes.”
“The grant is partly a result of recent knowledge condensed through a retrospective natural history study of LMNA-CMD via an international collaboration (the LMNA International Consortium). The new study will use valuable tools including Cure CMD's own CMDIR, OPALE-France patient registries, and will include access to patient biological material donated by Cure CMD’s most important partners: the LMNA-affected community.”
Rachel Alarez, Cure CMD Director of Operations adds "We are thrilled to announce the recipient of the co-funded grant with Muscular Dystrophy UK, whose mission is so closely aligned with ours.
Says Dr. Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK: “As muscle-wasting conditions are so rare, it’s important that we work together with others to boost research, and that’s why we are so proud of the LMNA-CMD partnership with Cure CMD.”
“Dr Bonne is a world expert and this project should take us a step forward in understanding why people with the condition experience such different symptoms, ultimately helping us to design better care, and one day, treatments.”
“We are so grateful to all the families who have worked so hard to raise money for this project and look forward to sharing the results.”
Cure CMD was founded in 2008 to advance research for treatments and a cure for congenital muscular dystrophy (CMD and, through engagement and support of the CMD community, to also improve the lives of those living with CMD. To date, the organization has funded more than $2 million in research, helped launch effective animal models, and has connected more than 2,000 affected individuals to a supportive, helpful community. Visit www.curecmd.org for more information.
MUSCULAR DYSTROPHY UK Muscular Dystrophy UK (MDUK) is the charity for the 70,000 people living with muscle-wasting conditions in the UK. The organization brings together people affected by more than 60 rare and very rare progressive muscle-weakening and wasting conditions. Since 1959, MDUK has been supporting families living with muscle-wasting conditions, providing vital information, advice, resources and support for people with these conditions, their families and the professionals who work with them.