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Myasthenia Gravis Rare Disease Network (MGNet)

The Myasthenia Gravis Rare Disease Network (MGNet) is a consortium of academic medical centers partnering with the Myasthenia Gravis Foundation of America and Conquer MG as well as collaborators in other research groups and industry. We are working together to enhance therapeutic development for this rare disease.

Diseases Studied

Myasthenia Gravis (MG) and its subtypes:

• Early-onset acetylcholine receptor (AChR) antibody (Ab) positive MG, which primarily affects women
• Late-onset AChR Ab positive MG with a disease bias towards men
• Paraneoplastic thymoma-associated MG
• Muscle specific kinase (MuSK) Ab positive
• AChR/MuSK antibody negative MG

Goals, Mission, & Vision

The goal of MGNet is to advance therapeutic development. To do so we have four specific aims:

• Improve the tools scientists and doctors have for performing clinical trials through rigorous monitoring of patients over time and evaluate better measures of how patients are doing.
• Identify objective measures (biomarkers) to show how the disease is responding to treatment and better predict treatment response or side effects, to improve the design of clinical trials and care of patients in clinical practice.
• Enhance the education of young investigators focusing their careers on rare diseases and specifically MG.
• Improve the awareness of scientists, physicians, and the public regarding the unique needs of patients with MG through training of clinician scientists and engagement of the patient and scientific communities in clinical research and therapeutic development.

Scholar Awards

Objectives

There is a critical need for investigators prepared to conduct clinical and translational research in myasthenia gravis (MG). The MGNet Scholar Program provides individualized training and mentorship in one of two tracks, Clinical Research or Biomarker Development Research.

Award Description

Myasthenia Gravis Rare Disease Network (MGNet)

The Myasthenia Gravis Rare Disease Network (MGNet) is a consortium of academic medical centers partnering with the Myasthenia Gravis Foundation of America and Conquer MG as well as collaborators in other research groups and industry. We are working together to enhance therapeutic development for this rare disease.

Diseases Studied

Myasthenia Gravis (MG) and its subtypes:

• Early-onset acetylcholine receptor (AChR) antibody (Ab) positive MG, which primarily affects women
• Late-onset AChR Ab positive MG with a disease bias towards men
• Paraneoplastic thymoma-associated MG
• Muscle specific kinase (MuSK) Ab positive
• AChR/MuSK antibody negative MG

Goals, Mission, & Vision

The goal of MGNet is to advance therapeutic development. To do so we have four specific aims:

• Improve the tools scientists and doctors have for performing clinical trials through rigorous monitoring of patients over time and evaluate better measures of how patients are doing.
• Identify objective measures (biomarkers) to show how the disease is responding to treatment and better predict treatment response or side effects, to improve the design of clinical trials and care of patients in clinical practice.
• Enhance the education of young investigators focusing their careers on rare diseases and specifically MG.
• Improve the awareness of scientists, physicians, and the public regarding the unique needs of patients with MG through training of clinician scientists and engagement of the patient and scientific communities in clinical research and therapeutic development.

Pilot and Feasibility Grants

Objectives

MGNet, a NIH-funded Rare Diseases Clinical Research Consortium (RDCRC) in partnership with the Myasthenia Gravis Foundation of America and Conquer MG, is seeking proposals for highly innovative investigations in early stages of development.

The priorities of the Pilot Grant Program are:

• Identification treatment responsive biomarkers
• Assess clinical measures and improve that enhance clinical trial readiness
• Evaluation of therapeutic interventions for one or more of the five distinct sub-types of MG

Pilot studies must be designed to access the resources of MGNet and focused on clinical and translational research, including fulfilling the definition of clinical and translational research and exclusion of animal studies.

Funding

The MGNet expects to fund up to two grants, each at a level of $75,000 (IDCs will be limited to 10% and included in the $75,000). Projects will be funded for 12 months. Selection among the applications received will be based on the recommendations of a review committee. A grant may be renewed for an additional one year with approval of interim report by the same review process and availability of funds. Demonstration of fulfillment of initial aims and justification for additional funding will be required. The request will be required a minimum of three months prior to the initial funding period completion.