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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.

Dissertation Grants

With support from the National Science Foundation, the American Educational Research Association (AERA) Grants Program seeks proposals for Dissertation Grants. The AERA Grants Program provides advanced graduate students with research funding and professional development and training. The program supports highly competitive dissertation research using rigorous quantitative methods to examine large-scale, education-related data. The aim of the program is to advance fundamental knowledge of relevance to STEM education policy, foster significant science using education data, promote equity in STEM, and build research capacity in education and learning. Since 1991, this AERA Program has been vital to both research and training at early career stages.

The Grants Program encourages the use of major data sets from multiple and diverse sources. It emphasizes the advanced statistical analysis of data sets from the U.S. Department of Education’s National Center for Education Statistics (NCES), the National Science Foundation (NSF), and other federal agencies. The program also supports studies using large-scale international data systems (e.g., PISA, PIRLS, or TIMMS) that benefit from U.S. federal government support. In addition, statewide longitudinal administrative data systems (SLDS) enhanced through federal grants are also eligible for consideration. The inclusion of federal or state administrative information that further expands the analytic capacity of the research is permissible. The thrust of the analysis needs to be generalizable to a national, state, or population or a subgroup within the sample that the dataset represents.

The Grants Program is open to field-initiated research and welcomes proposals that:

• develop or benefit from advanced statistical or innovative quantitative methods or measures;
• analyze more than one large-scale national or international federally funded data set, or more than one statewide longitudinal data system (SLDS) or incorporate other data enhancements;
• integrate, link, or blend multiple large-scale data sources; or
• undertake replication research of major findings or major studies using large-scale, federally supported or enhanced data.

The Grants Program encourages proposals across the life span and contexts of education and learning of relevance to STEM policy and practice. The research may focus on a wide range of topics, including but not limited to such issues as student achievement in STEM, analysis of STEM education policies, contextual factors in education, educational participation and persistence (pre-kindergarten through graduate school), early childhood education and development, postsecondary education, and the STEM workforce and transitions. Studies that examine issues of diversity, equity, and inclusion across STEM topics and/or for specific racial and ethnic groups, social classes, genders, or persons with disabilities are encouraged.

Award

• Award Component 1 - $27,500 Stipend
• Award Component 2 - AERA Research and Training Conference
• Award Component 3- AERA Annual Meeting Capstone Research Institute

The budget must include funds to attend the AERA Annual Meeting. The funds can be used for research-related expenses such as tuition, living expenses, travel to secure data enclaves or scholarly conferences, books, computer equipment, and other expenses directly related to conducting this research. AERA encourages cost sharing from universities in the form of tuition assistance, office space, university fees, and other expenses.