One‐year grant to support research related to a rare diseases

Research Areas for the 2020 MDBR Grant Program:

Adult Polyglucosan Body Disease (APBD)

Ataxia-Telangiectasia (A-T)

BPAN- A Neurodegeneration with Brain Iron Accumulation Disorder

CADASIL, Castleman Disease

Choroideremia

Congenital Hyperinsulinism (CHI)

Congenital Muscular Dystrophy (CMD)

Charcot Maire Tooth (CMT)

Cohen Syndrome (COH)

Nonsense Mutations in Cystic Fibrosis

Dyskeratosis Congenita & Telomere Biology Disorder

Fibrous Dysplasia/McCune Albright Syndrome

Fibrodysplasia Ossificans Progressiva (FOP)

Generalized Lymphatic Anomaly (GLA; a.k.a. lymphangiomatosis) and Gorham-Stout Disease (GSD)

Glut1 Deficiency Sundrome

Inclusion Body Myositis (IBM)

Lymphangioleiomyomatosis (LAM)

Mucopolysaccharidoses (MPS)

MPS Gene Spotlight

Mucopolysaccharidosis (MPS I)

Maple Syrup Urine Disease (MSUD)

Mitochondrial Complex 1 Deficiency Disorder - NUBPL

Neuroendocrine cell Hyperplasia of Infancy (NEHI)

Niemann Pick Type C (NPC)

Pitt Hopkins Syndrome (PTHS)

RASopathies, SETBP1 Disorder

Snyder-Robinson Syndrome (SRS)

STXBP1 Encephalopathy

TBCK Syndrome

All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA.

A one page letter of Interest (LOI), is due no later than Friday, September 18, 2020 by 8pm (EST)

Full proposals are by invitation only and are due no later than Monday, October 26, 2020 no later than 8pm (EST)

How to apply

A one-page Letter of Interest is to be submitted at: https://orphandiseasecenter.submittable.com/submit/170310/2020-million-dollar-bike-ride-pilot-grant-loi

Full applications:

- Cover Page/Checklist/Institutional Signature Page.
- NIH-style Biosketch with Other Support of PI and key personnel (5 pages max).
- Detailed Budget and Justification.
- Research Plan (5 pages max) and Bibliography (1 page max).
- Appendix (5 pages max of supplemental information