PATIENT RESOURCES AND CLINICAL TRIAL INFORMATION
Compassion. Connection. Hope.
At The Dion Foundation for Children with Rare Diseases, we recognize how deeply families are affected by LGMD2C and the urgent need for treatments. We are honored to be part of this journey toward progress and healing.
Our Role in the Clinical Trial
The Dion Foundation is a nonprofit organization that proudly provided financial support for the U.S. deployment of cohort #1 of Atamyo Therapeutics’ clinical trial.
However, it’s important to note that we are not involved in patient recruitment or enrollment and therefore cannot comment on, influence, or guarantee participation in the trial.
Where to Find Official Clinical Trial Information
For the most accurate and up-to-date information about Atamyo Therapeutics’ LGMD2C clinical trial, please refer to the official sources below:
🔗 Atamyo Contact Page — for questions directly related to the clinical trial
Our Mission & Advocacy
The Dion Foundation’s mission is to accelerate research, support drug development, and advocate for policies that improve the lives of children living with LGMD2C.
We continue to fund both pre-clinical and clinical-stage research, while also championing important legislation such as the Give Kids a Chance Act (H.R. 1262) — a bill designed to advance rare disease treatment access and innovation.
Learn more about our advocacy work here:
🔗 LGMD Advocacy & Legislative Action
JOIN OUR PATIENT REGISTRY
We know how important every update is.
If you wish to be notified when new information becomes available, please join our patient registry maintained by the Dion Foundation in compliance with HIPAA privacy policies.
Support the Mission
Your generosity helps us continue funding research and advocacy that bring hope to families affected by LGMD2C.
Donate Online:
🔗 www.thedionfund.org/donate-today
By Mail:
The Dion Foundation for Children with Rare Diseases, Inc.
PO Box 550011
Waltham, Massachusetts 02455
Checks payable to: The Dion Foundation
Federal Tax ID: 92-2280592
Our Commitment
We are so grateful for your compassion, patience, and commitment to this shared fight.
The Dion Foundation exists to amplify the voices of families living with LGMD2C and to ensure that promising treatments become accessible to all who need them.
With gratitude and hope,
— The Dion Foundation Team
www.thedionfund.org
LGMD 2C/R5 SARCOGLYCANOPATHY
PATIENT REGISTRY & CONTACT LIST
THE DION FOUNDATION IS CURRENTLY RECRUITING LGMD 2C/R5 PATIENTS TO JOIN OUR PATIENT CONTACT LIST
The purpose of this patient contact list is to help assist in the development of treatments for LGMD2C. Our goal for obtaining an accurate patient list includes:
Help researchers identify patients who quality for future clinical studies and trials
Share information regarding natural history studies that help researchers study LGMD2C progression
Share advocacy opportunities for patients/families afflicted with LGMD2C
Assist patients in finding the most up-to-date and disease specific clinical care
Share The Dion Foundation’s involvement in funding research
Provide patients and families with a care network of other patients and families afflicted with LGMD2C
diagnosis
+ care
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What is the role of genetic testing for muscular dystrophy (MDYS)?
Muscular dystrophy refers to a group of disorders characterised by progressive muscle weakness and loss of muscle tissue. In specific forms, other muscles—including respiratory muscles, cardiac smooth muscles, facial muscles and swallowing muscles—can also be affected.
Many forms of muscular dystrophy have overlapping clinical features making diagnosis difficult. Genetic testing has been proven to shorten the time to diagnosis and prevent misdiagnosis.
Accurate and early identification of affected individuals allows for improved clinical outcomes and the opportunity to participate in clinical trials.
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MUSCULAR DYSTROPHY ASSOCIATION
Located at more than 150 of the top health care institutions across the United States, MDA Care Centers and Affiliates serve as the nexus for expert clinical care and medical research. Our Care Center Network offers tens of thousands of appointments each year for individuals living with muscular dystrophy to access expert multidisciplinary care, clinical trials, and to connect with MDA and the neuromuscular community.
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PATIENT
INVOLVEMENT
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Participating in natural history studies and patient registries can help researchers gain further knowledge about ultra-rare diseases, such as LGMD sarcoglycanopathies.
Furthermore, natural history studies are designed to provide data for clinical trials to replace traditional “placebo” controlled studies.
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Muscular dystrophy patient advocacy groups have been among the most active in the rare disease drug development space. Notable achievements in the last decade include promulgating the first U.S. clinical research guidance, setting up registries and natural history studies, and investing in companies—some of which have brought potentially disease-modifying products to the market.
Patients’ voice is imperative to drug development: to support research, reform legislature, participate in clinical trial design, and encourage pharmaceutical industry that the patient community is ready for clinical studies.
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