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Displaying 1 - 12 of 12

ChANGE HD

Condition: Neurological Disorders / Huntington's Disease
Investigator: Ashwini Rao, EdD, OTR
Status: Currently Recruiting
Child to Adult Neurodevelopment in Gene Expanded Huntingtons Disease (ChANGE HD) HD has long been considered an adult-onset disease. However, research has shown that brain changes are evident before motor symptoms begin. In fact, the gene responsible for HD plays a role in brain development and is present throughout the lifespan. With gene knockdown therapy…
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Investigating Brain Changes in People with Cerebral Palsy

Condition: Neurological Disorders / Cerebral Palsy
Investigator: Jason Carmel, MD, PhD
Status: Currently Recruiting
A key part of the CP definition is that is a non-progressive brain injury. However, as people with CP age, their function often deteriorates. This study seeks to determine whether changes in the brain structure may result in function changes, as they do in other types of brain injuries.
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Cerebral Palsy Patient Registry

Condition: Neurological Disorders / Cerebral Palsy
Investigator: Jason Carmel, MD, PhD
Status: Currently Recruiting
The Weinberg Family Cerebral Palsy Center's (WFCPC) research team maintains a cerebral palsy (CP) patient registry of more than 1,000 enrolled patients. This registry contains information collected from children and adults with CP. The goal of the registry is to help us learn more about CP and neuromuscular diseases, to understand the quality of life…
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DfHD - Dance for Huntington's Disease

Condition: Neurological Disorders / Huntington's Disease
Investigator: Ashwini Rao, EdD, OTR
Status: Currently Recruiting
Dance is a complex activity involving the integration of rhythm, spatial patterns, synchronization to external stimuli and whole-body coordination. While regular exercise is often recommended for people with Huntington's Disease (HD), there are no studies examining the use of dance to improve balance and mobility and positively affect the quality of…
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SAGE HD-201

Condition: Neurological Disorders / Huntington's Disease
Investigator: Karen Marder, MD, MPH
Status: Closed
The DIMENSION Study is evaluating the safety and effect on cognitive symptoms of an investigational oral drug in adults with early Huntington's disease. Cognitive symptoms may include difficulty paying attention or trouble thinking through steps of an activity or complex problems. Symptoms may also include difficulty planning, remembering, and staying…
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Identifying Cerebral Palsy Pain Biomarkers

Condition: Neurological Disorders / Cerebral Palsy
Investigator: Joshua Hyman, MD
Status: Closed
Pain is a pervasive problem for persons with Cerebral Palsy (CP). The level to which many people with CP suffer from pain is mostly unknown and oftentimes difficult to determine. People with CP who are nonverbal or are unable to communicate cannot report their pain to their doctors or caregivers. We are doing this research study to better understand if the…
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A study for patients with spinal muscular atrophy using study drug, ISIS 396443 to test safety and effectiveness

Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Claudia Chiriboga, MD, MPH
Status: Closed
The purpose of this study is to evaluate the safety, tolerability and effectiveness of a study drug called ISIS-396443 when using it to treat children with later-onset spinal muscular atrophy (SMA). SMA is a neuromuscular disease that causes a degeneration (a breakdown) of the motor neurons in the spinal cord, resulting in a weakening of the muscles in…
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Tolerability, Safety, and Activity of study drug SRX246 in Irritable Subjects With Huntington's Disease

Condition: Neurological Disorders / Huntington's Disease
Investigator: Karen Marder, MD, MPH
Status: Closed
The purpose of this research study is to find out whether a new investigational drug called SRX246, which might be useful to Huntington's Disease (HD) patients who sometimes feel irritable, angry, or even aggressive, is well tolerated and safe when it is given two times a day by mouth at doses as high as 160 mg. As part of this study, we will use a…
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Brave Trial, A Clinical Research Study for Duchenne Muscular Dystrophy (DMD)

Condition: Pediatrics / Neuromuscular
Investigator: Darryl De Vivo, MD
Status: Closed
Our clinic is currently participating in the Brave Trial, a clinical research study for Duchenne muscular dystrophy (DMD). This study is being conducted to learn whether a study drug can improve motor functions in boys with DMD. If your child takes part, he'll receive at no cost: study drug or placebo, study-related procedures, reimbursement for…
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Flex CMT Muscle Cramps Study

Condition: Pediatrics / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
Moving forward in Charcot-Marie-Tooth disease. A clinical research study assessing the safety and effectiveness of an investigational product in people with Charcot-Marie-Tooth (CMT) disease experiencing muscle cramps is now enrolling. Participation is entirely voluntary. Contacting us does not mean that you are required to take part in this study. If you…
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A study for infants with pre-symptomatic Spinal Muscular Atrophy (SMA)

Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Darryl De Vivo, MD
Status: Closed
This study is for infants that have been genetically diagnosed with Spinal Muscular Atrophy (SMA) and are currently not showing any signs or symptoms of the disease. The purpose of this study is to see whether ISIS 396443 has any effects (good or bad) on infants with SMA (who do not yet show signs or symptoms of the disease) and to see if ISIS 396443 can…
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A study for male adolecents with muscular dystrophy using study drug Eteplirsen

Condition: Pediatrics / Neuromuscular
Investigator: Jacinda Sampson, MD
Status: Closed
The purpose of this study is to find out if eteplirsen can help people with the muscle disorder called Duchenne Muscular Dystrophy (DMD) and find out if eteplirsen is safe to take without causing too many side effects. Eteplirsen is an investigational (experimental) drug and can only be used in research studies. DMD is caused by a mutation (a change) in the…
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