Myotonic Dystrophy Foundation (MDF)
Our vision is a world with treatments and a cure for myotonic dystrophy. MDF is the leading global advocate helping patients and families navigate the DM disease process, and is often the first resource contacted by newly-diagnosed patients, their families, their social workers and their physicians around the world.
Myotonic Dystrophy Patients’ Group of Japan (DM-family)
Myotonic Dystrophy Patients’ Group of Japan, commonly known as DM-family, was established in 2016.
Until then, there had been no patient group exclusively for myotonic dystrophy in Japan. “I want to talk with other DM patients and their families. I hope my friends will join me.” This was what Akane Hatano, the founder of DM-family, was feeling when she attended a lecture by a group of doctors and learned that patient registry was necessary for the development of therapeutic drugs.
IDMC-13 Families Day: https://idmc13.dm-family.net/en/
Cure Myotonic Dystrophy UK Charity (Cure DM)
Cure DM is a UK Charity which supports those living with Myotonic Dystrophy, their families, and caregivers. All trustees and volunteers involved have personal experience of living with neuromuscular disease and are dedicated to improving quality of life for our loved ones. We have a focus on the often under-represented and overlooked childhood and congenital onset forms of the condition.
Late 2019 a major event happened in the domain of rare diseases: most of the European patient association involved in or dedicated to myotonic dystrophies decided to join their effort and federate in the first European patient association fully committed to Myotonic Dystrophies, called Euro-DyMA, representing roughly 100000 patients all across Europe.
Myotonic Dystrophy Support Group (MDSG)
Myotonic Dystrophy Support Group known as MDSG was the first support group founded to give support to only families with DM. that was 34 years ago. Amazing how the awareness of the condition has spread around the world, BUT not far enough. One of the aims of the UK MDSG is to spread awareness not just to families but to medical professionals.
Translational Genomics Group (Universidad de Valencia, Spain)
Our overall objective is to understand the mechanisms of pathogenesis in DM1 with a particular focus on muscle atrophy phenotypes and discover therapeutic targets and innovative treatments for patients. We use human cells, animal models, and bioinformatics to understand the contribution of miRNome alterations to the disease genesis, perform drug screenings, and evaluate the activity of Muscleblind enhancer drugs and therapeutics against novel targets. We are actively working in RNA-based drug development to achieve this, with our studies now in a preclinical state.