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Neuromuscular Disorders Topic Center

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 hall of posters for presentation at a professional conference
Conference Coverage
Golodirsen Eases Ambulatory, Pulmonary Function Decline in DMD
04/01/2024
Jolynn Tumolo
In patients with progressing Duchenne muscular dystrophy (DMD), golodirsen treatment over 6 years delayed loss of ambulation a median 2.4 years compared with matched controls.
In patients with progressing Duchenne muscular dystrophy (DMD), golodirsen treatment over 6 years delayed loss of ambulation a median 2.4 years compared with matched controls.
In patients with progressing...
04/01/2024
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
Jolynn Tumolo
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
Jolynn Tumolo
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
Jolynn Tumolo
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
Conference Coverage
In Patients With Spinal Muscular Atrophy, Higher Rate of Structural Brain Abnormalities Observed
10/11/2023
Jolynn Tumolo
Structural brain abnormalities are more common in patients with spinal muscular atrophy (SMA) compared with typically developing peer controls.
Structural brain abnormalities are more common in patients with spinal muscular atrophy (SMA) compared with typically developing peer controls.
Structural brain abnormalities...
10/11/2023
Neurology
News
Guideline Addresses Respiratory Management of Patients With Neuromuscular Weakness
04/18/2023
Jolynn Tumolo
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
Jolynn Tumolo
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

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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
Jolynn Tumolo
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
Jolynn Tumolo
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
Jolynn Tumolo
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
Jolynn Tumolo
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
Jolynn Tumolo
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
Jolynn Tumolo
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
Brionna Mendoza
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
Fremanezumab Shows Positive Results for Pediatric Use in Phase 3 Trial
News
Fremanezumab Shows Positive Results for Pediatric Use in Phase 3 Trial
08/08/2024
In July, Teva announced positive results in a phase 3 trial of migraine medication Ajovy (fremanezumab) for children and adolescents, making it the first anti-CGRP (calcitonin gene-related peptide) monoclonal antibody to be effective in this...
In July, Teva announced positive results in a phase 3 trial of migraine medication Ajovy (fremanezumab) for children and adolescents, making it the first anti-CGRP (calcitonin gene-related peptide) monoclonal antibody to be effective in this...
In July, Teva announced positive...
08/08/2024
Neurology
brain waves
News
High-Dose Statins May Not Benefit Patients With Mild Ischemic Stroke
08/08/2024
Compared with moderate-dose therapy, high-dose statin therapy in patients with mild ischemic stroke did not decrease the risk of stroke recurrence and was associated with an increased risk of intracranial hemorrhage over 12 months.
Compared with moderate-dose therapy, high-dose statin therapy in patients with mild ischemic stroke did not decrease the risk of stroke recurrence and was associated with an increased risk of intracranial hemorrhage over 12 months.
Compared with moderate-dose...
08/08/2024
Neurology
An illustration of a healthcare provider conducting a telehealth appointment
News
Teleneurology Visits Acceptable to Patients Across Neurologic Conditions
08/05/2024
Regardless of their neurologic condition, patients rated outpatient teleneurology appointments as highly acceptable, according to a study published in the Journal of Neurology.
Regardless of their neurologic condition, patients rated outpatient teleneurology appointments as highly acceptable, according to a study published in the Journal of Neurology.
Regardless of their neurologic...
08/05/2024
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
A person walks down a boarded path with sunlight and trees in the background
News
Backward Walking Speed Reserve Reflects Disease Severity in Patients With MS
07/30/2024
Backward walking speed reserve, or the difference between an individual’s preferred and maximal backward walking speed, was associated with disease severity in patients with multiple sclerosis (MS).
Backward walking speed reserve, or the difference between an individual’s preferred and maximal backward walking speed, was associated with disease severity in patients with multiple sclerosis (MS).
Backward walking speed reserve,...
07/30/2024
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
A middle-aged male running
News
Moderate Physical Activity May Mitigate ALS Risk for Male Patients
07/25/2024
Male individuals who engage in moderate physical activity and fitness may lower their risk for amyotrophic lateral sclerosis (ALS) later in life, according to study findings published in Neurology.
Male individuals who engage in moderate physical activity and fitness may lower their risk for amyotrophic lateral sclerosis (ALS) later in life, according to study findings published in Neurology.
Male individuals who engage in...
07/25/2024
Neurology
Pitolisant Receives FDA Approval for Pediatric Patients With Narcolepsy
News
Pitolisant Receives FDA Approval for Pediatric Patients With Narcolepsy
07/23/2024
The US Food and Drug Administration (FDA) recently approved Wakix (pitolisant) for the treatment of excessive daytime sleepiness in pediatric patients 6 years and older with narcolepsy, US maker Harmony Biosciences announced.
The US Food and Drug Administration (FDA) recently approved Wakix (pitolisant) for the treatment of excessive daytime sleepiness in pediatric patients 6 years and older with narcolepsy, US maker Harmony Biosciences announced.
The US Food and Drug...
07/23/2024
Neurology
Chronic Loneliness Associated With Hiked Stroke Risk in Older Adults
News
Chronic Loneliness Associated With Hiked Stroke Risk in Older Adults
07/22/2024
Chronic loneliness was associated with higher stroke risk, independent of other factors, according to recent prospective cohort study results published in eClinicalMedicine. 
Chronic loneliness was associated with higher stroke risk, independent of other factors, according to recent prospective cohort study results published in eClinicalMedicine. 
Chronic loneliness was...
07/22/2024
Neurology
a hand breaks a wooden human figurine in half
News
Atogepant Reduces Migraine Days in Patients With Pain Medication Overuse
07/18/2024
The calcitonin gene-related peptide inhibitor atogepant reduced monthly migraine and headache days, as well as days using pain medication, in patients with chronic migraine with and without acute medication overuse.
The calcitonin gene-related peptide inhibitor atogepant reduced monthly migraine and headache days, as well as days using pain medication, in patients with chronic migraine with and without acute medication overuse.
The calcitonin gene-related...
07/18/2024
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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