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Neuromuscular Disorders Topic Center

News
Guideline Addresses Respiratory Management of Patients With Neuromuscular Weakness
04/18/2023
A clinical guideline for the respiratory management of patients with neuromuscular weakness was recently released by the American College of Chest Physicians in the journal CHEST.
A clinical guideline for the respiratory management of patients with neuromuscular weakness was recently released by the American College of Chest Physicians in the journal CHEST.
A clinical guideline for the...
04/18/2023
Neurology
News
Huntington Disease Compromises Striatal Neurons Differently
02/13/2023
Huntington disease appears to affect two distinct cell populations in the striatum differently, according to a study published online in Nature Communications by neuroscientists at the Massachusetts Institute of Technology.
Huntington disease appears to affect two distinct cell populations in the striatum differently, according to a study published online in Nature Communications by neuroscientists at the Massachusetts Institute of Technology.
Huntington disease appears to...
02/13/2023
Neurology
News
In Parkinson Disease, Inflammation May Play Causative Role
12/28/2022
A growing body of evidence suggests inflammation could play a causative role in Parkinson disease, according to a special supplement to the Journal of Parkinson’s Disease.
A growing body of evidence suggests inflammation could play a causative role in Parkinson disease, according to a special supplement to the Journal of Parkinson’s Disease.
A growing body of evidence...
12/28/2022
Neurology
News
For Some Infants With Spinal Muscular Atrophy, Earlier Intervention May Be Warranted
11/15/2022
New research has called into question the “watchful waiting” strategy for infants born with spinal muscular atrophy (SMA) and multiple copies of the SMN2 gene.
New research has called into question the “watchful waiting” strategy for infants born with spinal muscular atrophy (SMA) and multiple copies of the SMN2 gene.
New research has called into...
11/15/2022
Neurology
Yingcheng Liu, PhD student, MIT CSAIL.
Q&As
Using Remote Radio Wave Technology to Improve Parkinson Disease Patient Outcomes
10/17/2022
Yingcheng Liu talks to Neurology Learning Network about an exciting new technology: a home device that uses radio waves to detect and analyze the movements of individuals living with Parkinson disease.
Yingcheng Liu talks to Neurology Learning Network about an exciting new technology: a home device that uses radio waves to detect and analyze the movements of individuals living with Parkinson disease.
Yingcheng Liu talks to Neurology...
10/17/2022
Neurology
News
Eli-Cel Gene Therapy Approved for Early, Active Cerebral Adrenoleukodystrophy
10/12/2022
The FDA granted accelerated approval to elivaldogene autotemcel, also known as eli-cel (Skysona), to slow the progression of neurologic dysfunction in boys 4 through 17 years of age with early, active cerebral adrenoleukodystrophy (CALD).
The FDA granted accelerated approval to elivaldogene autotemcel, also known as eli-cel (Skysona), to slow the progression of neurologic dysfunction in boys 4 through 17 years of age with early, active cerebral adrenoleukodystrophy (CALD).
The FDA granted accelerated...
10/12/2022
Neurology
News
COVID-19 May Accelerate Brain Aging, Increase Neurological Risk
08/23/2022
The consequences of COVID-19 infection appear to include a predisposition to irreversible neurological conditions, an increased likelihood of stroke, and the potential for chronic brain lesions that can lead to microbleeds, according to a...
The consequences of COVID-19 infection appear to include a predisposition to irreversible neurological conditions, an increased likelihood of stroke, and the potential for chronic brain lesions that can lead to microbleeds, according to a...
The consequences of COVID-19...
08/23/2022
Neurology
News
FDA Advisory Committee Recommends Approval of Adrenoleukodystrophy Treatment
07/01/2022
An FDA advisory panel unanimously recommended the approval of the investigational gene therapy elivaldogene autotemcel (eli-cel) for the treatment of early active cerebral adrenoleukodystrophy in pediatric patients.
An FDA advisory panel unanimously recommended the approval of the investigational gene therapy elivaldogene autotemcel (eli-cel) for the treatment of early active cerebral adrenoleukodystrophy in pediatric patients.
An FDA advisory panel...
07/01/2022
Neurology
The Neurologist Is In podcast.
Podcasts
The Neurologist Is In, Episode 10, Part 1: Providing Interdisciplinary Patient Care With Neuromuscular Specialist Dr Madhu Mona Soni
06/20/2022
In this episode, Dr Rachel Salas, MD, MEd, sits down with Madhu Mona Soni, MD, FAAN, to discuss exciting developments in neuromuscular neurology, the power of interdisciplinary patient care, and more!
In this episode, Dr Rachel Salas, MD, MEd, sits down with Madhu Mona Soni, MD, FAAN, to discuss exciting developments in neuromuscular neurology, the power of interdisciplinary patient care, and more!
In this episode, Dr Rachel...
06/20/2022
Neurology
Quiz
Is Time to Motor Loss Function in Spinal Muscular Atrophy Predictable?
07/21/2021
True or False: Time to loss of motor function in Spinal Muscular Atrophy is predictable.
True or False: Time to loss of motor function in Spinal Muscular Atrophy is predictable.
True or False: Time to loss of...
07/21/2021
Neurology

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Parkinson Disease Risk Elevated in Patients With Upper GI Mucosal Damage
09/30/2024
A history of upper gastrointestinal mucosal damage is associated with a 76% increased risk of subsequent Parkinson disease (PD), according to a study published online in JAMA Network Open.
A history of upper gastrointestinal mucosal damage is associated with a 76% increased risk of subsequent Parkinson disease (PD), according to a study published online in JAMA Network Open.
A history of upper...
09/30/2024
Neurology
a scientist works with biomarkers in a lab
News
DYNE-251 for DMD Yields Promising Biomarker, Functional Results in Early-Stage Trial
09/10/2024
Patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping demonstrated unprecedented dystrophin expression and functional improvement with the investigational therapeutic DYNE-251.
Patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping demonstrated unprecedented dystrophin expression and functional improvement with the investigational therapeutic DYNE-251.
Patients with Duchenne muscular...
09/10/2024
Neurology
The US Food and Drug Administration headquarters sign.
News
DMD Gene Therapy Indication Expanded to Include Boys 4 and Older
08/01/2024
The US FDA approved an indication expansion for the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) to include patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are nonambulatory and 4...
The US FDA approved an indication expansion for the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) to include patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are nonambulatory and 4...
The US FDA approved an...
08/01/2024
Neurology
FDA Approves Efgartigimod Alfa/Hyaluronidase for CIDP Treatment
News
FDA Approves Efgartigimod Alfa/Hyaluronidase for CIDP Treatment
07/29/2024
VYVGART Hytrulo (efgartigimod alfa/hyaluronidase) was recently approved by the Food and Drug Administration (FDA) for the treatment of adults with chronic inflammatory demyelinating polyneuropathy (CIDP). 
VYVGART Hytrulo (efgartigimod alfa/hyaluronidase) was recently approved by the Food and Drug Administration (FDA) for the treatment of adults with chronic inflammatory demyelinating polyneuropathy (CIDP). 
VYVGART Hytrulo (efgartigimod...
07/29/2024
Neurology
Doctors examine brain scans
News
Drug Trial Shows Cognitive Impact of Huntington Disease, but Analysts Express Caution
06/24/2024
Scores on a cognitive battery showed a statistically significant difference in patients with Huntington disease and healthy control subjects at baseline in a phase 2 study involving the dalzanemdor, an investigational NMDA receptor positive...
Scores on a cognitive battery showed a statistically significant difference in patients with Huntington disease and healthy control subjects at baseline in a phase 2 study involving the dalzanemdor, an investigational NMDA receptor positive...
Scores on a cognitive battery...
06/24/2024
Neurology
A teddy bear against a blue background wearing a mask and stethoscope.
News
Anavex 2-73 Shows Improvement in Pediatric Rett Syndrome But Misses Key Endpoint
02/02/2024
Children with Rett syndrome who received Anavex 2-73 (blarcamesine) showed improvement on the Rett Syndrome Behaviour Questionnaire (RSBQ) but failed to meet a trial key endpoint for Clinical Global Impression – Improvement (CGI-I).
Children with Rett syndrome who received Anavex 2-73 (blarcamesine) showed improvement on the Rett Syndrome Behaviour Questionnaire (RSBQ) but failed to meet a trial key endpoint for Clinical Global Impression – Improvement (CGI-I).
Children with Rett syndrome who...
02/02/2024
Neurology
A person completes a puzzle of a profile of a head, with a piece missing from where the brain is located.
News
ME/CFS and Fibromyalgia May Fall Along Same Illness Spectrum, CSF Analysis Suggests
01/16/2024
Myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) and fibromyalgia do not appear to be distinct conditions, according to an analysis of cerebrospinal fluid (CSF) proteomes
Myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) and fibromyalgia do not appear to be distinct conditions, according to an analysis of cerebrospinal fluid (CSF) proteomes
Myalgic...
01/16/2024
Neurology
An illustrated picture shows two cartoon doctors, one holding up a magnifying glass to a cartoon brain.
News
Gene Therapy Improves Motor Function but Misses Primary Endpoint in Duchenne Muscular Dystrophy Trial
11/21/2023
The single-dose gene therapy Elevidys (delandistrogene moxeparvovec-rokl) increased motor function in children ages 4 through 7 years with Duchenne muscular dystrophy compared with placebo.
The single-dose gene therapy Elevidys (delandistrogene moxeparvovec-rokl) increased motor function in children ages 4 through 7 years with Duchenne muscular dystrophy compared with placebo.
The single-dose gene therapy...
11/21/2023
Neurology
News
Guideline Addresses Respiratory Management of Patients With Neuromuscular Weakness
04/18/2023
A clinical guideline for the respiratory management of patients with neuromuscular weakness was recently released by the American College of Chest Physicians in the journal CHEST.
A clinical guideline for the respiratory management of patients with neuromuscular weakness was recently released by the American College of Chest Physicians in the journal CHEST.
A clinical guideline for the...
04/18/2023
Neurology
News
Huntington Disease Compromises Striatal Neurons Differently
02/13/2023
Huntington disease appears to affect two distinct cell populations in the striatum differently, according to a study published online in Nature Communications by neuroscientists at the Massachusetts Institute of Technology.
Huntington disease appears to affect two distinct cell populations in the striatum differently, according to a study published online in Nature Communications by neuroscientists at the Massachusetts Institute of Technology.
Huntington disease appears to...
02/13/2023
Neurology
neurology brain stress
News
Targeted Lucidity Reactivation May Help Ease Narcolepsy-Related Nightmares
11/18/2024
Results from a pilot study of patients with narcolepsy provide proof-of-concept evidence that a procedure called targeted lucidity reactivation can be adapted for narcolepsy-related nightmares.
Results from a pilot study of patients with narcolepsy provide proof-of-concept evidence that a procedure called targeted lucidity reactivation can be adapted for narcolepsy-related nightmares.
Results from a pilot study of...
11/18/2024
Neurology
A doctor examines a patient who has a head injury from soccer
News
Concussion With Depression Worsens Symptoms, Brain Connectivity
11/15/2024
The comorbidity of depression and sports-related concussion amplifies symptoms of both conditions, according to study findings published in Brain Research.
The comorbidity of depression and sports-related concussion amplifies symptoms of both conditions, according to study findings published in Brain Research.
The comorbidity of depression...
11/15/2024
Psych Congress Network
Semaglutide May Lower Risk of Alzheimer Disease in Patients With Type 2 Diabetes
News
Semaglutide May Lower Risk of Alzheimer Disease in Patients With Type 2 Diabetes
11/05/2024
Patients with type 2 diabetes and obesity prescribed semaglutide had a 40% to 70% lower risk of a first-time Alzheimer disease (AD) diagnosis compared with patients taking other antidiabetic medications, according to a study published in...
Patients with type 2 diabetes and obesity prescribed semaglutide had a 40% to 70% lower risk of a first-time Alzheimer disease (AD) diagnosis compared with patients taking other antidiabetic medications, according to a study published in...
Patients with type 2 diabetes...
11/05/2024
Psych Congress Network
Alone or Together, CBT and Modafinil Improve MS-Related Fatigue
News
Alone or Together, CBT and Modafinil Improve MS-Related Fatigue
11/01/2024
The wake-promoting drug modafinil, CBT, and a combination of both were associated with comparable, significant improvement in fatigue at 12 weeks in patients with MS and problematic fatigue.
The wake-promoting drug modafinil, CBT, and a combination of both were associated with comparable, significant improvement in fatigue at 12 weeks in patients with MS and problematic fatigue.
The wake-promoting drug...
11/01/2024
Neurology
Gene therapy
News
Concerns Linger After Gene Therapy Shows Long-Term Benefit for Cerebral Adrenoleukodystrophy
10/28/2024
Elivaldogene autotemcel (eli-cel) gene therapy for cerebral adrenoleukodystrophy (CALD) helped patients maintain neurological function, but also my have caused hematologic malignancies in some patients.
Elivaldogene autotemcel (eli-cel) gene therapy for cerebral adrenoleukodystrophy (CALD) helped patients maintain neurological function, but also my have caused hematologic malignancies in some patients.
Elivaldogene autotemcel...
10/28/2024
Neurology
Patient with family
News
During Recovery, Stroke Patients Benefit from Supportive Social Networks
10/24/2024
Large networks of family and friends are beneficial for blood pressure improvement among patients who experienced a stroke, according to a study published online in the International Journal of Cerebrovascular Disease and Stroke. “In aging...
Large networks of family and friends are beneficial for blood pressure improvement among patients who experienced a stroke, according to a study published online in the International Journal of Cerebrovascular Disease and Stroke. “In aging...
Large networks of family and...
10/24/2024
Neurology
International Stroke Burden Saw Hike Over Last 30 Years
News
International Stroke Burden Saw Hike Over Last 30 Years
10/22/2024
International stroke burden has increased from 1990 to 2021, according to recent findings from a global burden of disease (GBD) study published in The Lancet Neurology.
International stroke burden has increased from 1990 to 2021, according to recent findings from a global burden of disease (GBD) study published in The Lancet Neurology.
International stroke burden has...
10/22/2024
Neurology
Hands holding a head in profile with an illustrated heart over brain
News
Antiseizure Meds May Increase Cardiovascular Risk in Older Adults With Epilepsy
10/21/2024
A lifetime history of epilepsy is associated with new-onset cardiovascular events in older adults, according to study results published in JAMA Neurology.
A lifetime history of epilepsy is associated with new-onset cardiovascular events in older adults, according to study results published in JAMA Neurology.
A lifetime history of epilepsy...
10/21/2024
Neurology
FDA approval stamp
News
FDA Approves Infusion Treatment for Advanced Parkinson Disease in Adults
10/18/2024
AbbVie’s VYALEV (foscarbidopa and foslevodopa), the first 24-hour continuous subcutaneous infusion of levodopa-based therapy for managing motor fluctuations in adults with advanced Parkinson disease (PD), approved by the FDA.
AbbVie’s VYALEV (foscarbidopa and foslevodopa), the first 24-hour continuous subcutaneous infusion of levodopa-based therapy for managing motor fluctuations in adults with advanced Parkinson disease (PD), approved by the FDA.
AbbVie’s VYALEV (foscarbidopa...
10/18/2024
Neurology
Investigational Drug Improves Motor Function in Patients With SMA
News
Investigational Drug Improves Motor Function in Patients With SMA
10/16/2024
Apitegromab, an investigational muscle-targeted therapy, improved motor function in patients with spinal muscular atrophy (SMA), late-stage biopharmaceutical company Scholar Rock recently announced.
Apitegromab, an investigational muscle-targeted therapy, improved motor function in patients with spinal muscular atrophy (SMA), late-stage biopharmaceutical company Scholar Rock recently announced.
Apitegromab, an investigational...
10/16/2024
Neurology

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Alzheimer's Disease CME Series

Early Alzheimer’s Disease: Caregiver Connections, Shared Decision Making, and Patient Resources
11/19/2020
Early Alzheimer’s Disease: Caregiver Connections, Shared Decision Making, and Patient Resources To view accreditation information/faculty disclosures for the program, click 
Early Alzheimer’s Disease: Caregiver Connections, Shared Decision Making, and Patient Resources To view accreditation information/faculty disclosures for the program, click 
Early Alzheimer’s Disease:...
11/19/2020
Neurology
Early Alzheimer’s Disease: Caregiver Connections, Shared Decision Making, and Patient Resources
11/19/2020
Early Alzheimer’s Disease: Caregiver Connections, Shared Decision Making, and Patient ResourcesOnline Form - PBHLN-NLN Alzheimers Video Series
Early Alzheimer’s Disease: Caregiver Connections, Shared Decision Making, and Patient ResourcesOnline Form - PBHLN-NLN Alzheimers Video Series
Early Alzheimer’s Disease:...
11/19/2020
Neurology
Early Alzheimer’s Disease: Caregiver Connections, Shared Decision Making, and Patient Resources
11/19/2020
Early Alzheimer’s Disease: Caregiver Connections, Shared Decision Making, and Patient ResourcesTo claim credit for the program, click HERE for the evaluation. You will need to log in or create an account with NACCME, and then click...
Early Alzheimer’s Disease: Caregiver Connections, Shared Decision Making, and Patient ResourcesTo claim credit for the program, click HERE for the evaluation. You will need to log in or create an account with NACCME, and then click...
Early Alzheimer’s Disease:...
11/19/2020
Neurology
Emerging Treatments for Early Alzheimer Disease: Efficacy and Safety Data
11/17/2020
Emerging Treatments for Early Alzheimer Disease: Efficacy and Safety Data To view accreditation information/faculty disclosures for the program, click 
Emerging Treatments for Early Alzheimer Disease: Efficacy and Safety Data To view accreditation information/faculty disclosures for the program, click 
Emerging Treatments for Early...
11/17/2020
Neurology
Emerging Treatments for Early Alzheimer’s Disease: Efficacy and Safety Data
11/17/2020
Emerging Treatments for Early Alzheimer’s Disease: Efficacy and Safety Data Online Form - PBHLN-NLN Alzheimers Video Series
Emerging Treatments for Early Alzheimer’s Disease: Efficacy and Safety Data Online Form - PBHLN-NLN Alzheimers Video Series
Emerging Treatments for Early...
11/17/2020
Neurology
Emerging Treatments for Early Alzheimer’s Disease: Efficacy and Safety Data
11/17/2020
Emerging Treatments for Early Alzheimer’s Disease: Efficacy and Safety DataTo claim credit for the program, click HERE for the evaluation. You will need to log in or create an account with NACCME, and then click on the evaluation...
Emerging Treatments for Early Alzheimer’s Disease: Efficacy and Safety DataTo claim credit for the program, click HERE for the evaluation. You will need to log in or create an account with NACCME, and then click on the evaluation...
Emerging Treatments for Early...
11/17/2020
Neurology
Emerging Treatments for Early Alzheimer’s Disease: Mechanisms of Action
11/12/2020
Emerging Treatments for Early Alzheimer’s Disease: Mechanisms of Action To view accreditation information/faculty disclosures for the program, click 
Emerging Treatments for Early Alzheimer’s Disease: Mechanisms of Action To view accreditation information/faculty disclosures for the program, click 
Emerging Treatments for Early...
11/12/2020
Neurology
Early Alzheimer’s Disease Overview: Burden of Disease, Quality-of-Life Impacts, and Pathophysiology
11/12/2020
Early Alzheimer’s Disease Overview: Burden of Disease, Quality-of-Life Impacts, and Pathophysiology Online Form - PBHLN-NLN Alzheimers Video Series
Early Alzheimer’s Disease Overview: Burden of Disease, Quality-of-Life Impacts, and Pathophysiology Online Form - PBHLN-NLN Alzheimers Video Series
Early Alzheimer’s Disease...
11/12/2020
Neurology
Claim-Emerging Treatments for Early Alzheimer’s Disease: Mechanisms of Action
11/12/2020
Emerging Treatments for Early Alzheimer’s Disease: Mechanisms of ActionTo claim credit for the program, click HERE for the evaluation. You will need to log in or create an account with NACCME, and then click on the evaluation button...
Emerging Treatments for Early Alzheimer’s Disease: Mechanisms of ActionTo claim credit for the program, click HERE for the evaluation. You will need to log in or create an account with NACCME, and then click on the evaluation button...
Emerging Treatments for Early...
11/12/2020
Neurology
Current Treatment Limitations and Clinical Needs in Early Alzheimer’s Disease
11/10/2020
Current Treatment Limitations and Clinical Needs in Early Alzheimer’s Disease To view accreditation information/faculty disclosures for the program, click 
Current Treatment Limitations and Clinical Needs in Early Alzheimer’s Disease To view accreditation information/faculty disclosures for the program, click 
Current Treatment Limitations...
11/10/2020
Neurology

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