Advances in DMD Care: Evaluating a Gene Therapy’s Efficacy and Safety

Guest: Kelly Gwathmey, MD ALS can take up to a year to diagnose, but researchers are hard at work trying to find ways to minimize diagnostic delays and errors. One such solution is the Rapid Access ALS Clinic, where patients were diagnosed an average of two months sooner. Here to talk about this work is Dr. Kelly Gwathmey, Professor of Neurology at the Virginia Commonwealth University School of Medicine who presented a session on this topic at the 2024 American Association of Neuromuscular and Electrodiagnostic Medicine Annual Meeting.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Stacy Finkbeiner, PhD Guest: Bill Cote To gain insights into how tardive dyskinesia (TD) can impact the physical, psychological, social, and professional lives of patients, the IMPACT-TD Registry incorporated feedback from patients and caregivers to create an effective questionnaire. These patient-reported measures are key to the overall management of TD as they enable us to better understand how TD affects each individual. To learn more about the patient-reported outcomes from the IMPACT-TD Registry, Dr. Charles Turck speaks with Dr. Stacy Finkbeiner and Mr. Bill Cote. Dr. Finkbeiner is US Medical Director at Teva Pharmaceuticals, and Mr. Cote is the Senior Director of the National Organization for Tardive Dyskinesia.

Host: Ashley Baker, MSN, PMHNP Guest: Stacy Finkbeiner, PhD The IMPACT-TD Registry is an ongoing, real-world observational study that aims to better understand the multidimensional impacts of tardive dyskinesia (TD) on patients’ lives. To achieve this goal, the IMPACT-TD Scale was developed and used by clinicians involved with the study, and now the results from the interim analysis are available. Joining psychiatric nurse practitioner Ashley Baker to share the key findings on TD’s impact from the clinician perspective is Dr. Stacy Finkbeiner, U.S. Medical Director at Teva Pharmaceuticals.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Andrew J. Cutler, MD Guest: Michelle Scargle, MD Tardive dyskinesia is quite common as up to 20 percent of patients may develop this hyperkinetic movement disorder. But despite its prevalence, there are many barriers that can keep us from screening patients for tardive dyskinesia. To learn more about these barriers and how we can overcome them to optimize our screening approach, Dr. Charles Turck speaks with Drs. Andrew Cutler and Michelle Scargle. Dr. Cutler is a Clinical Associate Professor in the Department of Psychiatry and Behavioral Sciences at the SUNY Upstate Medical University in Syracuse, New York, and Dr. Scargle is the Chief Psychiatrist at Concord Health in Clearwater, Florida.

Host: Ashley Baker, MSN, PMHNP Guest: Richard Jackson, MD The IMPACT-TD scale enables us to evaluate the effects of tardive dyskinesia across four key domains: psychological/psychiatric, social, physical, and vocational/educational/recreational. By using this assessment tool, we can not only gain a deeper understanding of how our patients are impacted by tardive dyskinesia, but it can also foster better communication and treatment outcomes. To learn more, psychiatric nurse practitioner Ashley Baker speaks with Dr. Richard Jackson, Associate Clinical Professor at the Oakland Beaumont University School of Medicine and an Assistant Clinical Professor at the University of Michigan School of Medicine.

Guest: John F. Brandsema, MD Between 60 and 70 percent of patients with Duchenne muscular dystrophy (DMD) also have a diagnosable neurobehavioral phenotype like autism, ADHD, and anxiety. Here to talk about these common neurobehavioral issues in patients with DMD and how we can manage them is Dr. John Brandsema, Neuromuscular Section Head at the Children’s Hospital of Philadelphia.

Guest: John F. Brandsema, MD As patients with Duchenne muscular dystrophy (DMD) age, their treatment regimens, considerations, and goals change. That’s why Dr. John Brandsema is here to talk about how we can best treat and support patients with DMD throughout their entire care journey. Dr. Brandsema is the Neuromuscular Section Head at the Children’s Hospital of Philadelphia.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: John F. Brandsema, MD The gene therapy delandistrogene moxeparvovec was recently approved for patients with Duchenne muscular dystrophy (DMD) who are at least 4 years old. This expanded approval is based on the efficacy and safety results from the EMBARK trial. Joining Dr. Charles Turck to break down those key findings and the implications of this advancement in DMD treatment is Dr. John Brandsema. Dr. Brandsema is the Neuromuscular Section Head at the Children’s Hospital of Philadelphia, where one of the clinical trials for this gene therapy took place.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Jasmohan Bajaj, MD Around 50 percent of people with cirrhosis have hepatic encephalopathy, which means some kind of brain dysfunction. As these patients age, it gets more and more difficult to differentiate between hepatic encephalopathy and dementia, which is why two recent studies sought to uncover how many patients with dementia have undiagnosed cirrhosis. According to the findings, the rate of undiagnosed cirrhosis and hepatic encephalopathy could be as high as 10‒13 percent. Joining Dr. Charles Turck to share further details about the studies, findings, and implications is Dr. Jasmohan Bajaj, Professor of Medicine in the Division of Gastroenterology, Hepatology, and Nutrition at Virginia Commonwealth University.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Dr. Robert J. Shmookler Reis, PhD Guest: Dr. Srinivas Ayyadevara, PhD Numerous factors could predispose patients to the progression of mild cognitive impairment or dementia and Alzheimer’s disease. Fortunately, a recent study found that a cholesterol-lowering medication called ezetimibe might help reduce the risk of Alzheimer’s disease progression and related dementias. To learn about the methods and key findings of the study, join Dr. Charles Turck as he takes a deep dive with Dr. Robert Reis, Professor and Researcher at the University of Arkansas for Medical Sciences’ Institute on Aging College of Medicine and Research Career Scientist at the Central Arkansas Veterans Healthcare System’s Geriatric Research, Education and Clinical Center, and Dr. Srini Ayyadevara, Associate Professor at the UAMS Institute on Aging College of Medicine and is a Research Health Scientist at the Central Arkansas VA’s GREC Center.