# New Directions in Biology and Disease of Skeletal Muscle

> **NIH NIH R13** · NORTHWESTERN UNIVERSITY · 2020 · $15,000

## Abstract

PROJECT SUMMARY
This is a new proposal requesting partial support for "The New Directions in Biology and Disease of Skeletal
Muscle," to be held July 6-9, 2020. The meeting will convene at the New York Marriott Marquis in Times
Square. The “New Directions” initiated in 2004, and this meeting has taken place every other year, making
the 2020 meeting the 9th in the series. The goal of this meeting is to promote translational science for
neuromuscular disease, especially the muscular dystrophies. In 2004, at the time of the first meeting, there
were no FDA approved drugs for neuromuscular disease and a limited pipeline with limited interest from the
pharmaceutical industry and biotech. With the purpose of this meeting to bring together stakeholders including
academic laboratories, small and large pharmaceutical interests, and patient/family advocacy groups, this
meeting has been instrumental in promoting the identification and translation of new targets for neuromuscular
disease. This meeting was developed in response to the MD Care Act, which recognized a need for a
neuromuscular disease focused meeting with specific emphasis on target identification and translation of basic
discovery into clinical trials for those with neuromuscular disease. The New Directions meeting differs from
other muscle biology-related meetings because of inclusion of industry and academic attendees as well as
heavy emphasis on trainee participation. Exon skipping drugs were approved for Duchenne Muscular
Dystrophy (DMD) and Spinal Muscular Atrophy (SMA) in 2016 and 2017. Gene therapy for SMA in 2019 with
approved as onasmemnogene abeparvovec, and there are multiple ongoing micro-dystrophin gene therapy
trials for DMD. Based on this progress and on feedback from participants at the 2018 meeting, this next
meeting will expand to other forms of inherited muscular diseases and focus on novel mechanisms for these
disorders.

## Key facts

- **NIH application ID:** 10070741
- **Project number:** 1R13AR078066-01
- **Recipient organization:** NORTHWESTERN UNIVERSITY
- **Principal Investigator:** Elizabeth M McNally
- **Activity code:** R13 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $15,000
- **Award type:** 1
- **Project period:** 2020-09-01 → 2022-02-28

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10070741

## Citation

> US National Institutes of Health, RePORTER application 10070741, New Directions in Biology and Disease of Skeletal Muscle (1R13AR078066-01). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10070741. Licensed CC0.

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