DM-Family is an association of myotonic dystrophy patients which is active in the following areas.
＊The Japanese fiscal year runs from April to the following March.
To Contribute to DM Research as Patients
DM Patient Registry Surpasses 1,300 Participants!
The number of participants in the Japanese DM patient registry ‘Remudy’ has now exceeded 1,300. This amounts to 13% of the estimated 10,000 patients in Japan.The Remudy registry meets all of the essential and recommended elements of the Naardén dataset, which was agreed by countries around the world in 2009. Its registration has been achieved through the efforts of doctors and patients.
DM-Family continues its activities to promote the importance of the registry to patients and their families in order to increase the number of registrants.
The Japanese national registry for myotonic dystrophy (DM) is jointly operated by the National Center of Neurology and Psychiatry (NCNP) and the University of Osaka.
As of March 2025
A Series of Lectures to Learn from Basic Research to Therapeutics
In October 2024, DM-Family hosted a webinar on genes, muscle cells, and genome editing.
Patients who ask, "Will my muscles recover?" should also understand the importance of basic research.
In the webinar, we listened to clear explanations about genes and muscle cells and learned that the development of siRNA drugs targeting skeletal and cardiac muscle is progressing. We also learned that research is advancing in creating and expanding iPS cells from the skin and blood cells of DM patients, differentiating them into muscle cells, and using them for treatment.
This was a valuable opportunity for patients and their families to learn about the cutting-edge research taking place in Japan.
Introducing Companies Developing Treatments from Around the World
It is important for patients and their families to be aware of the companies developing therapeutic drugs that may lead to future clinical research. To support this, DM-Family has included the names and logos of these companies on the Myotonic Dystrophy Type 1 Drug Development Status Chart to provide the latest information for patients and families in Japan.
As of April 2025
Presentation of Development Products from Overseas Pharmaceutical Companies
In August 2024, DM-Family hosted online presentations from Dyne Therapeutics, Inc., in December, PepGen Inc., and in January, Avidity Biosciences, Inc., regarding the therapeutic drugs these companies are developing. Each company sincerely and clearly explained the mechanisms of their respective therapeutic drugs as well as the interim results of clinical trials with the help of interpreters. Japanese patients and their families learned about the current state of drug development and felt hopeful for the future.
Exhibiting at Academic Conferences
DM-Family participates in academic conferences, such as the Annual Meeting of the Japanese Society of Neurology, to provide detailed information about myotonic dystrophy to medical professionals, especially those outside the neuromuscular specialty. These exhibitions also serve as valuable opportunities for pharmaceutical companies to engage with DM patients and their families. The exhibition is a collaborative effort involving patients and their families, and DM-Family values the friendships and trust that have developed through this shared experience.
To Enrich the Hearts and Minds of Patients and Their Families
Sharing a Heartfelt Patient Journey
Patients and families living with intractable diseases find it difficult to talk openly about their experiences. Over time, with the support of family members and physicians, they begin to share their stories. Listening to these personal journeys is a deeply meaningful and enriching experience -- a wonderful time to spend.
Public Presentation by a Member: Dedication Shown by Three-Hour One-Way Visits to a Specialized Hospital
At a hybrid seminar held in Hiroshima Prefecture, DM-Family member Noriko Ooyoshi spoke about the benefits of visiting a specialized hospital.
Noriko’s younger sister is a DM patient with mild intellectual disability. The sisters live in a rural area where there are no specialized hospitals nearby. Wondering, “Isn’t there something I can do for my sister?” Noriko was inspired to join DM-Family and decided to take her sister to a specialized hospital.
The trip to the hospital, which takes three hours each way, is not easy for her and her sister.
At the specialized hospital, they were able to receive treatment for several complications that their local hospital could not address.
Noriko says that she and her sister often talk about wanting to live happily while coping with DM, which has been diagnosed as "incurable," and about their hope to make a difference in the world by participating in patient association activities.
At the seminar, Noriko shared the importance of having enthusiasm and courage with the many patients and family members in attendance.
International DM Awareness Day: Tea Party in Saitama
On September 15 2024, DM patients, their families, and supporters from all over Japan gathered in Saitama for a tea party.
Kousuke Ogawa began his speech by saying: ‘What Japanese patients and their families are doing is not known in Western countries, where Japanese language is not understood! By posting photos of patients, families, and supporters on the English website of DM-Family, we have finally managed to share some of this.” He also said, “Let's all take a photo together to commemorate this day!” —lifting the spirits of the participants.
At the party, participants listened to a short lecture about DM from a specialist, and one of the members also performed a magic show. In addition, during a workshop entitled “Stretching You Can Do While Sitting”, each table was assigned a physiotherapist who carefully guided participants through stretching exercises.
The time for face-to-face discussion flew by, and as the party drew to a close, participants were reluctant to say goodbye. There was a growing sense of unity and determination among the patients and their families.
To Raise Public Awareness About DM
Hybrid Seminar in Hiroshima: Everything from Care to Drug Therapies!
DM-Family held a hybrid seminar in Hiroshima on July 28, 2024. In just three hours, we covered the basic knowledge that patients, families and supporters should have, such as “the basics of daily care”, “how to access social services”, “registries and natural history studies”, and “the current state of treatment development”.
Participating in the International Myotonic Dystrophy Consortium (IDMC-14)
From April 9 to 13 2024, the 14th International Myotonic Dystrophy Consortium (IDMC-14) was held in Nijmegen, in the Netherlands, and organized by Radboud University and Maastricht University Medical Center. Members from DM Family also participated in the conference.
In addition to basic research and clinical sessions, there were also sessions on patient-centered topics such as quality of life, rehabilitation, and issues specific to female patients.
At this first in-person meeting since the COVID -19 pandemic, DM-Family members were able to engage in direct discussions and interactions with pharmaceutical companies and patient groups from various countries.
Participating in “Euro-DyMA Pharma's Day”
In the early morning of April 9, before the start of IDMC-14, Representatives of DM-Family attended “Euro-DyMA Pharma's Day”, an invitation-only event organized by the EU patient group alliance Euro-DyMA.
PepGen, AMO Pharma, Arthex Biotech, Avidity Biosciences, and Dyne Therapeutics explained how their respective products work. Companies that have already conducted clinical trials discussed their results.
Lecture on Drug Loss from the Perspective of Patients and Their Families
"Drug loss"—a situation caused by the fact that the development of treatments (such as clinical trials) takes place overseas but not in Japan—has become a Japanese social issue. DM-Family’s Managing Director, Midori Senoo, delivered a speech on behalf of patients and their families at the National Hospital Medical Association and the Muscular Dystrophy Medical Research Association.
Drug loss is a real problem for patients with rare diseases. Midori Senoo pointed out that, while several companies are conducting clinical trials overseas, opportunities for such trials are still limited in Japan.
She also expressed her commitment to providing patients and their families with accurate information about clinical trials.
Lecture by a Current PMDA Official: Japan’s Drug Review—The Fastest in the World!
In November 2024, a current employee of the Pharmaceuticals and Medical Devices Agency (PMDA) gave a lecture on the role of the PMDA and how drug reviews are conducted. While PMDA is also responsible for safety measures and addressing health damages, its lecture focused solely on drug reviews to benefit patients and their families. The lecture covered a wide range of topics, including the clinical trial process, rules for clinical trials, the PMDA’s role, and the fact that the basis of evaluation is a "comparison". It provided valuable insights into the challenges of developing safe and effective medicines.
It was also highlighted that “the current issue is no longer the lengthy review period but rather drug loss”.
Attending the Global Alliance for DM Awareness, Connecting with Friends around the World
DM-Family is a member of the Global Alliance, a group of 60 organizations worldwide dedicated to myotonic dystrophy.
Under the slogan "September 15 is International Myotonic Dystrophy Awareness Day," we are committed to raising awareness of myotonic dystrophy among patients, families, and people from all walks of life.
DM-Family aims to overcome this disease not only in Japan, but also in cooperation with other countries around the world, so that patients can contribute to society.