HOW TO TAKE ACTION

Advocacy

HOW TO TAKE ACTION

Advocacy

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Advocacy

Advocacy plays a major role in preparing for successful clinical trials, especially for a rare disease. Individuals with dysferlinopathy have many opportunities to support advancement in research, reform legislature, contribute to improved clinical experiences, and demonstrate to drug developers that the community is ready for trials. There are large goals for the entire LGMD community to accomplish as well as critical disease specific goals.

Advocacy plays a major role in preparing for successful clinical trials, especially for a rare disease.

What are Registries and Why are they Important?

One of the most fundamental and urgent needs that patients express is the need to connect with the researchers who are focused on their subtype and to be accounted for in preparation for clinical trial recruitment. Patient registries can act as a central hub where the community can work to fill the needs of patients to connect and so much more.

The purpose of the registry determines how it will be designed and utilized. A well-designed registry can serve as a critical resource for improving the lives of patients and accelerating progress and trial recruitment. A registry can:

  • Collect “Natural History” Data
  • Analyze patient reported outcomes
  • Match patients with studies and/or trials/accelerate trial recruitment
  • Patient family networking
  • Identify clinical care needs
  • Record impact ways the disease impacts quality of life

The Dysferlin Registry is a registry of people genetically diagnosed with dysferlinopathy, also referred to as LGMD2B, LGMDR2, Miyoshi Myopathy 1, managed by the Jain Foundation. To learn more about the Dysferlin Registry go to: dysferlinregistry.jain-foundation.org, or contact us at patients@jain-foundation.org.

Ways you can make a difference

Participate in Trials/Studies

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Join the Dysferlin Registry

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Connect with the LGMD Awareness Foundation

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Register for the International LGMD Patient Conference

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