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Neuromuscular Disorders Topic Center

News
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 hall of posters for presentation at a professional conference
Conference Coverage
Golodirsen Eases Ambulatory, Pulmonary Function Decline in DMD
04/01/2024
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
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
Conference Coverage
In Patients With Spinal Muscular Atrophy, Higher Rate of Structural Brain Abnormalities Observed
10/11/2023
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

News
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
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
Tolebrutinib Delays Disability Progression in Nonrelapsing Secondary Progressive MS
News
Tolebrutinib Delays Disability Progression in Nonrelapsing Secondary Progressive MS
10/15/2024
Tolebrutinib delayed the time to onset of 6-month confirmed disability progression compared with placebo in patients with nonrelapsing secondary progressive MS, according to results presented at the European Committee for Treatment and...
Tolebrutinib delayed the time to onset of 6-month confirmed disability progression compared with placebo in patients with nonrelapsing secondary progressive MS, according to results presented at the European Committee for Treatment and...
Tolebrutinib delayed the time to...
10/15/2024
Neurology
An orange prescription container with generic pills spilling out in front of it
News
Masked Taper Plus CBT for Insomnia Effective in Benzodiazepine Discontinuation
10/10/2024
A masked drug taper combined with cognitive behavioral therapy for insomnia (CBTI) were associated with improved benzodiazepine receptor agonist discontinuation in patients receiving treatment for insomnia
A masked drug taper combined with cognitive behavioral therapy for insomnia (CBTI) were associated with improved benzodiazepine receptor agonist discontinuation in patients receiving treatment for insomnia
A masked drug taper combined...
10/10/2024
Psych Congress Network

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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