# Pilot Feasibility Core

> **NIH NIH U54** · UNIVERSITY OF IOWA · 2020 · $97,496

## Abstract

Abstract
CMT affects 1/2500 people and there are no proven treatments that modify the disease. Clinical
and basic research in inherited peripheral neuropathies is expanding rapidly on many fronts.
Next generation sequencing enables the identification of novel genetic causes such that > 90
genes causing CMT neuropathy have now been identified, many by sites within the INC.
Rational therapeutic approaches for the most common subtypes of CMT are being actively
pursued or have even already developed. The INC needs to rapidly respond to new
opportunities and needs that result from novel discoveries in fields such as genetic diagnosis,
gene editing, cell biology, or outcome measures in CMT. To this end we propose a
Pilot/Feasibility Core to support single- or multi-site projects that will advance the diagnosis,
clinical trial readiness, management, and/or treatment of CMT subtypes. Approved projects will
typically be approved for a single year, with a budget of $25,000 to $50,000. Funds will be
available to support two Pilot/Feasibility projects per year. Junior INC investigators and senior
investigators wishing to expand their research in new directions will be encouraged to apply.
Evaluating Pilot/Feasibility applications and monitoring the projects will be the responsibility of
the Pilot/Feasibility Core, which will be directed by Dr. Mario Saporta (the site-PI of the clinical
University of Miami site and Dr. Alex Rossor (from our UCL site). They were the first two
scholar/trainees of the INC 10 years ago, and are now recognized as independent investigators
in our field. Core membership includes expertise in COAs, genetic diagnosis, and therapeutic
development. Members include the INC leadership - Drs. Shy, Scherer, Herrmann, Reilly,
Züchner, and Burns, as well as Drs. Finkel (an expert in neuromuscular trials) and Svaren (a
member of our External Advisory Committee, and an expert in biomarker development) and
representatives from two PAGS, Amy Gray (CMTA) and Dr. Amanda Haidet-Phillips (MDA).

## Key facts

- **NIH application ID:** 10004179
- **Project number:** 5U54NS065712-13
- **Recipient organization:** UNIVERSITY OF IOWA
- **Principal Investigator:** MICHAEL E. SHY
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $97,496
- **Award type:** 5
- **Project period:** 2009-09-30 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10004179

## Citation

> US National Institutes of Health, RePORTER application 10004179, Pilot Feasibility Core (5U54NS065712-13). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10004179. Licensed CC0.

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