US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

About Us

Our mission is to empower people living with neuromuscular diseases to live longer, more independent lives.

MDA Awards $300,000 to Streamline Gathering of Data for ALS Research

Project aims to implement a fundamental change in practice that could provide people with ALS a simple way to contribute to research that can lead to treatments and cures

CHICAGO, June 15, 2017 – The Muscular Dystrophy Association has announced the award of a clinical research network grant (CRNG) to Michael Benatar, M.D., Ph.D., at the University of Miami Miller School of Medicine in Florida, and Jonathan Katz, M.D., at California Pacific Medical Center in San Francisco, to support the Clinical Procedures To Support Research (CAPTURE) project, which aims to implement the “ALS Toolkit” within the Epic Electronic Health Record System. The ALS Toolkit provides a mechanism to systematize collection of clinical data so that it can also be used for research. The two-year award totaling $300,000 will support work conducted through the Clinical Research in ALS and Related Disorders for Therapy Development (CReATe) consortium, with the goal of lessening the burden on people with ALS who wish to participate in clinical research studies.

Throughout the course of the disease, people with ALS require input and assistance from multiple health care professionals. Because of this, multidisciplinary clinics such as MDA ALS Care Centers are a key resource for ongoing medical management and care. Visits to care centers take several hours, and a substantial amount of clinical data (such as neurological examination results, quantification of respiratory muscle strength and motor function assessments) are routinely collected — much of it identical to the assessments performed at specialized research visits. However, data from these two types of visits are typically captured separately, necessitating separate appointments for people who, depending on the stage of their disease, may experience profound weakness, disability and difficulty associated with travel.

With increasing penetration of the electronic medical record into academic medical centers and the recently established collaboration between Benatar and Katz with Epic, a team will now develop and implement a novel ALS Toolkit (set of “smart forms”) within the electronic medical record system that will enable clinicians to collect standardized data that can serve both clinical and research purposes.

“MDA is excited to support this effort to integrate research data collection into the standard visits patients have with their physicians — making it easier for people with ALS to participate in research,” said MDA Scientific Program Officer Amanda M. Haidet-Phillips, Ph.D. “The work being done by Drs. Benatar and Katz seeks to reduce the barriers that prevent some people with ALS from participating in research, and will make it easier for doctors to collect information that can be used for research purposes to gain a better understanding of ALS and its subtypes, develop biomarkers and accelerate therapy development. The hope is that making it easier for both patients and doctors to participate in research will increase involvement and speed progress.”

The project involves completion of the development of the ALS Toolkit in partnership with Epic, after which it will be piloted at the University of Miami and California Pacific Medical Center. Approximately 100 people with ALS will be enrolled for the testing phase of the toolkit at these two sites.

“The clinic is the most important venue for regularly seeing and evaluating ALS patients. In addition, valuable data is routinely collected and entered into electronic health records at every appointment, at every ALS Center across the country,” Benatar said. “To date, no one has taken the steps to structure this regular flow of data, or to combine them in a single database. Instead, we have costly research protocols that collect the same information, requiring an ongoing duplication of work, while attempts to transfer clinically collected data into a research database requires extra steps of duplicating data entry — once into the medical record and once into a research database.”

The new system represents an opportunity to fundamentally change current practice, and is expected to increase efficiency and reduce costs.

“Integration of a mechanism to collect research-useful data through a clinical workflow within the electronic health record offers a huge opportunity not just to increase efficiency, but to transform the power of clinically collected data to support the engine of research,” Katz said.

Researchers will be empowered to use clinical data for research purposes, and clinicians who currently are working in isolation in single centers will be able to take part in a wider research community.

MDA has dedicated more than $363 million to ALS research and support services since 1950. Including five ALS research grants announced in March, and the recent award to Benatar, MDA currently is funding 44 active ALS grants with a total funding commitment of $11.4 million. This year, MDA is funding 150 different research projects around the world.

About the Muscular Dystrophy Association
MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at

Roxan Triolo Olivas
MDA Vice President
Public Relations and Community Programs
(520) 529-5305