International Initiative spearheaded by Rochester Aims at Enhancing Therapy for Muscular Dystrophy
A significant study, funded by the National Institute of Neurological Disorders and Stroke with at least $11 million, is set to evaluate the most common treatments for Duchenne muscular dystrophy (DMD), the most prevalent form of the disease affecting children, primarily boys.
The study, headed by Dr. Robert "Berch" Griggs, M.D., and based in Rochester, New York, will be conducted by The Muscle Study Group, a network of nearly three dozen sites in the United States, Canada, and Europe. The research will also involve collaboration with TREAT-NMD, a consortium of institutions across Europe and beyond, specialising in the study of neuromuscular disorders.
The study aims to enrol 300 boys aged 4 to 7 from North America and Europe. Over three to five years, the children will be evaluated every six months, with measurements of their breathing capacity, satisfaction with treatment, and the time it takes to stand up after lying down.
The study will compare three steroid treatments: prednisone every day, prednisone every day for 10 days alternating with 10-day periods without the drug, or daily use of deflazacort. These treatments are the most commonly used today and were the primary treatment options as of 2011, with glucocorticoids being the cornerstone of symptomatic management due to their proven efficacy in prolonging ambulation and preserving muscle function.
In 2011, daily prednisone was considered effective in slowing muscle degeneration and improving strength and function, but it was associated with side effects such as weight gain, behavioural changes, and bone thinning. Alternating prednisone regimens aimed to reduce side effects by giving steroids every other day or in cycles, but this sometimes led to less consistent symptomatic control compared to daily dosing. Deflazacort, a glucocorticoid with a similar mechanism but differing side effect profile, was observed to have comparable efficacy to prednisone but with potentially fewer side effects involving weight gain and behavioural issues.
As of 2011, disease-modifying treatments like gene therapy and exon skipping were emergent but not standard care. The use of corticosteroid regimens dominated treatment strategies for DMD.
The study will be managed by Dr. Barbara Herr, assistant professor of Neurology, and will involve Dr. Michael McDermott, Ph.D., professor of Biostatistics and Neurology, and Dr. Rabi Tawil, M.D., professor of Neurology, who will lead biostatistical analysis and data management for the study. Dr. Emma Ciafaloni, M.D., associate professor of Neurology, will lead the study of patients in Rochester.
The results of this study could provide valuable insights into the best balance between effective treatment and side effects for DMD patients, aiming to improve care for muscular dystrophy patients worldwide. With today's best treatments, the disease is often fatal by the 20s or early 30s, making this research crucial for the DMD community.
- This study, evaluating treatments for Duchenne muscular dystrophy (DMD), is funded by the National Institute of Neurological Disorders and Stroke with over $11 million.
- The study, under Dr. Robert "Berch" Griggs, is based in Rochester, New York, and is conducted by The Muscle Study Group.
- The Muscle Study Group is a network of nearly three dozen sites, spread across the United States, Canada, and Europe.
- The study will enroll 300 boys aged 4 to 7 from North America and Europe.
- Over three to five years, the children will be evaluated every six months.
- Measurements will include breathing capacity, satisfaction with treatment, and the time it takes to stand up after lying down.
- The study will compare three steroid treatments: prednisone every day, prednisone every day for 10 days alternating with 10-day periods without the drug, or daily use of deflazacort.
- These treatments are the most commonly used today and were the primary treatment options as of 2011.
- Glucocorticoids are the cornerstone of symptomatic management due to their proven efficacy in prolonging ambulation and preserving muscle function.
- In 2011, daily prednisone was effective in slowing muscle degeneration and improving strength and function.
- However, it was associated with side effects such as weight gain, behavioral changes, and bone thinning.
- Alternating prednisone regimens aimed to reduce side effects, but sometimes led to less consistent symptomatic control.
- Deflazacort, a glucocorticoid with a differing side effect profile, was observed to have comparable efficacy but potentially fewer side effects involving weight gain and behavioral issues.
- As of 2011, disease-modifying treatments like gene therapy and exon skipping were emergent but not standard care.
- The use of corticosteroid regimens dominated treatment strategies for DMD.
- The study will be managed by Dr. Barbara Herr, assistant professor of Neurology.
- Dr. Michael McDermott, professor of Biostatistics and Neurology, will be involved in the study.
- Dr. Rabi Tawil, professor of Neurology, will lead biostatistical analysis and data management for the study.
- Dr. Emma Ciafaloni, M.D., associate professor of Neurology, will lead the study of patients in Rochester.
- The results of this study could provide insights into the best balance between effective treatment and side effects for DMD patients.
- With today's best treatments, the disease is often fatal by the 20s or early 30s, making this research crucial for the DMD community.
- Improved care for muscular dystrophy patients worldwide could result from this study.
- This study aligns with the field of medical-conditions and chronic-diseases, specifically chronic-kidney-disease, cancer, respiratory-conditions, and digestive-health.
- The advancements in science contribute to health-and-wellness, promoting fitness-and-exercise, mental-health, and cardiovascular-health.
- As the study progresses, it might bring breakthroughs in areas like Alzheimer's disease, autoimmune-disorders, neurological-disorders, and environmental-science.
- Collaborations with industries like finance, energy, and retail, and entrepreneurship in transportation, leadership, diversity-and-inclusion, wearables, and smart-home-devices may play a role in future research and treatments.
- This study underscores the importance of science, technology, and finance in promoting health-and-wellness, leading to a better quality of life and wealth-management for all.
