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UCLA Health – Southern California’s Frontlines of FSHD Research

Home / FSHD Research / UCLA Health – Southern California’s Frontlines of FSHD Research

Posted on: Sep 11, 2020

Perry Shieh, MD PhD

Nestled into a basement in an outpatient building at UCLA Health’s Westwood hospitals is UCLA Neurology, home to ground-breaking FSHD Research.   UCLA Health already ranks as the #1 hospital in California, and 4th nationally according to the U.S. News & World Report’s 2020 rankings, but it is also part of an elite network of research institutions in the FSHD Clinical Trial Research Network dedicated to better understanding FSHD and finding a treatment by 2025 to help the families suffering with the illness. 

Led by Dr. Perry Shieh, in recent years UCLA has been a clinical trial site for some of the most promising potential FSHD medicines and information-gathering studies. In 2017-2019 UCLA worked with Acceleron Pharma to conduct the ACE-083 study investigating if a myostatin inhibitor could increase muscle mass and strength. In 2018, UCLA joined ten other clinical trial sites across the U.S. and Europle for the Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD (ReSolve) study –  the largest longitudinal study measuring 220 genetically confirmed FSHD patients over 18+ months and seeking to validate two novel clinical outcome assessments including the Reachable Workspace leveraging Microsoft’s Xbox Kinect and the FSHD Timed Get-up and Go from a laying down position. In 2019, UCLA partnered with Fulcrum Therapeutics to investigate if Losmapimod could reduce the expression of DUX4 in FSHD patient’s muscles in an ongoing study that was recently extended due to COVID-19.  Dr. Shieh also leads his team of researchers in many other clinical trials beyond FSHD including the studies that led to the first FDA approved medicines for Spinal Muscular Atrophy and works on several studies investigating potential treatments for Duchenne Muscular Dystrophy. 

In addition to research, Dr. Shieh also treats patients in the Southern California community working to meet their neurological needs.  Weekly UCLA hosts a Muscular Dystrophy Association (MDA)  clinic with the goal of providing wrap-around neurological services to patients. During clinic days patients can meet with a coordinator from the MDA, a neurological physical therapist, orthotist and Dr. Shieh. As a research hospital, part of the patient experience at UCLA is also serving to educate neurological fellows and residents. Often during clinic a fellow will meet with patients as part of the comprehensive services. This offers patients an opportunity to educate medical professionals about the FSHD experience. 

Regardless of whether patients are interested in participating in trials themselves, Dr. Perry Shieh will patiently walk patients through recent research studies and explain why he is hopeful for a future treatment for FSHD in the near future. 

To schedule an appointment at UCLA, please contact 310-794-1195. Telehealth appointments are also available due to COVID-19.

This is a sponsored blog post by UCLA Health as part of their 2020 Los Angeles Walk & Roll Platinum Sponsorship Partnership.

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