Clinical Research Areas

At the Daniel B. Drachman Division of Neuromuscular Medicine, we are dedicated to mounting a comprehensive and united effort against diseases affecting the motor neurons, peripheral nerves and muscles. The division's team of experts includes clinicians and scientists who work collaboratively to investigate the genetic and molecular basis of neuromuscular disorders, such as muscular dystrophies, amyotrophic lateral sclerosis (ALS), peripheral neuropathy, and other complex conditions. Their research aims to develop novel therapies that enhance patient outcomes and quality of life. 

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Peripheral Neuropathy and Nerve Regeneration

The Neuromuscular Division's research program includes investigations into peripheral neuropathy, a common condition that affects the nerves outside of the brain and spinal cord, leading to numbness, tingling, pain and weakness in the hands and feet. Researchers at the Merkin Peripheral Neuropathy and Nerve Regeneration Center are studying the mechanisms that underlie peripheral neuropathy and developing new therapies that can alleviate symptoms and improve patients' quality of life. 

Amyotrophic Lateral Sclerosis (ALS)

Another area of focus for the division's research program is ALS, a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to muscle weakness and eventually paralysis. Researchers in the Packard Center are working to better understand the underlying causes of ALS and develop new treatments that can slow or halt disease progression. They are also investigating the role of genetics and epigenetics in ALS, with the goal of developing personalized therapies that target specific genetic mutations.

Muscle Diseases

The neuromuscular division also has an active program in diagnosis of diseases that primarily affect skeletal muscle including genetic and acquired myopathies. The Muscular Dystrophy Association Clinic researchers are studying the underlying mechanisms of muscle dysfunction and fibrosis and are developing therapies that may slow the disease progression or improve symptoms in individuals with muscle disease. With the aid of new classes of RNA and gene directed therapies, they aim to bring new treatments to individuals with muscular dystrophies and related conditions.

Collaboration across disciplines is a key aspect of the division's research program, as they work to integrate findings to better understand the complex mechanisms of neuromuscular diseases and develop effective treatments.

Charcot-Marie-Tooth (CMT) Disease

The Charcot-Marie-Tooth Center is part of the Inherited Neuropathy Consortium (INC), a group of academic medical centers, patient support organization, and clinical research resources dedicated to conducting clinical research in different forms of CMT and improving patient care. Our center participates in ongoing studies coordinated by the INC. Our patients who might benefit from and are eligible for clinical research studies are offered an opportunity to participate. Studies may include natural history, genetic and disease biomarker studies.

With our strong commitment to advancing scientific knowledge, we aim to achieve groundbreaking discoveries in neuromuscular research. Our ultimate goal is to provide renewed hope and better care for individuals impacted by these complex conditions.