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Neuromuscular Disorders Topic Center

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News
FDA Approves Copper Histidinate Treatment for Pediatric Menkes Disease
01/19/2026
Brionna Mendoza
The US Food and Drug Administration (FDA) approved a copper histidinate (Zycubo) injection as the first treatment for Menkes disease in pediatric patients.
The US Food and Drug Administration (FDA) approved a copper histidinate (Zycubo) injection as the first treatment for Menkes disease in pediatric patients.
The US Food and Drug...
01/19/2026
Neurology
Two doctors review research data on a computer in a lab
News
Deramiocel Meets Key Endpoints in Phase 3 HOPE-3 Duchenne Trial
12/05/2025
Brionna Mendoza
Deramiocel, an investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD), met its primary and secondary endpoints in the phase 3 HOPE-3 trial, according to Capricor Therapeutics.
Deramiocel, an investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD), met its primary and secondary endpoints in the phase 3 HOPE-3 trial, according to Capricor Therapeutics.
Deramiocel, an investigational...
12/05/2025
Neurology
US FDA sign
News
FDA Approves Gene Therapy for Spinal Muscular Atrophy in Patients Aged 2 Years and Older
12/02/2025
Brionna Mendoza
The FDA granted approval to onasemnogene abeparvovec-brve (Itvisma) on November 24, 2025, extending gene therapy to children older than 2, teens, and adults with spinal muscular atrophy (SMA)
The FDA granted approval to onasemnogene abeparvovec-brve (Itvisma) on November 24, 2025, extending gene therapy to children older than 2, teens, and adults with spinal muscular atrophy (SMA)
The FDA granted approval to...
12/02/2025
Neurology
Pop quiz written on a blackboard with white chalk
Quiz
Delandistrogene Moxeparvovec for Duchenne Muscular Dystrophy
10/22/2025
Brionna Mendoza
Test your knowledge with this quiz!
Test your knowledge with this quiz!
Test your knowledge with this...
10/22/2025
Neurology
An illustrated picture shows two cartoon doctors, one holding up a magnifying glass to a cartoon brain.
News
Gene Therapy Slows Huntington Disease Progression in Phase 1/2 Trial
10/06/2025
Jolynn Tumolo
Patients with Huntington disease who received a high dose of the gene therapy AMT-130 experienced 75% less disease progression after 36 months compared with a propensity score-matched external cohort that did not receive the treatment.
Patients with Huntington disease who received a high dose of the gene therapy AMT-130 experienced 75% less disease progression after 36 months compared with a propensity score-matched external cohort that did not receive the treatment.
Patients with Huntington disease...
10/06/2025
Neurology
two researchers look at computer screen in lab medical research
News
Apitegromab Improves Motor Function in Children With SMA
09/24/2025
Jolynn Tumolo
Treatment with the fully human monoclonal antibody apitegromab was associated with statistically significant improvements in motor function scores compared with placebo in patients with type 2 or type 3 spinal muscular atrophy (SMA).
Treatment with the fully human monoclonal antibody apitegromab was associated with statistically significant improvements in motor function scores compared with placebo in patients with type 2 or type 3 spinal muscular atrophy (SMA).
Treatment with the fully human...
09/24/2025
Neurology
A teddy bear against a blue background wearing a mask and stethoscope.
News
Phase 2 Trial Finds Risdiplam Effective and Safe in Newborns With SMA
08/21/2025
Jolynn Tumolo
Infants with presymptomatic spinal muscular atrophy (SMA) treated with risdiplam as early as 16 days of age had better functional and survival outcomes at 12 and 24 months compared with untreated infants with SMA.
Infants with presymptomatic spinal muscular atrophy (SMA) treated with risdiplam as early as 16 days of age had better functional and survival outcomes at 12 and 24 months compared with untreated infants with SMA.
Infants with presymptomatic...
08/21/2025
Neurology
News
Parkinson Disease Risk Elevated in Patients With Upper GI Mucosal Damage
09/30/2024
Jolynn Tumolo
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
Jolynn Tumolo
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
Jolynn Tumolo
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

Migraine CME Series

News

fda building sign
News
FDA Approves Copper Histidinate Treatment for Pediatric Menkes Disease
01/19/2026
Brionna Mendoza
The US Food and Drug Administration (FDA) approved a copper histidinate (Zycubo) injection as the first treatment for Menkes disease in pediatric patients.
The US Food and Drug Administration (FDA) approved a copper histidinate (Zycubo) injection as the first treatment for Menkes disease in pediatric patients.
The US Food and Drug...
01/19/2026
Neurology
Two doctors review research data on a computer in a lab
News
Deramiocel Meets Key Endpoints in Phase 3 HOPE-3 Duchenne Trial
12/05/2025
Brionna Mendoza
Deramiocel, an investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD), met its primary and secondary endpoints in the phase 3 HOPE-3 trial, according to Capricor Therapeutics.
Deramiocel, an investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD), met its primary and secondary endpoints in the phase 3 HOPE-3 trial, according to Capricor Therapeutics.
Deramiocel, an investigational...
12/05/2025
Neurology
US FDA sign
News
FDA Approves Gene Therapy for Spinal Muscular Atrophy in Patients Aged 2 Years and Older
12/02/2025
Brionna Mendoza
The FDA granted approval to onasemnogene abeparvovec-brve (Itvisma) on November 24, 2025, extending gene therapy to children older than 2, teens, and adults with spinal muscular atrophy (SMA)
The FDA granted approval to onasemnogene abeparvovec-brve (Itvisma) on November 24, 2025, extending gene therapy to children older than 2, teens, and adults with spinal muscular atrophy (SMA)
The FDA granted approval to...
12/02/2025
Neurology
An illustrated picture shows two cartoon doctors, one holding up a magnifying glass to a cartoon brain.
News
Gene Therapy Slows Huntington Disease Progression in Phase 1/2 Trial
10/06/2025
Jolynn Tumolo
Patients with Huntington disease who received a high dose of the gene therapy AMT-130 experienced 75% less disease progression after 36 months compared with a propensity score-matched external cohort that did not receive the treatment.
Patients with Huntington disease who received a high dose of the gene therapy AMT-130 experienced 75% less disease progression after 36 months compared with a propensity score-matched external cohort that did not receive the treatment.
Patients with Huntington disease...
10/06/2025
Neurology
two researchers look at computer screen in lab medical research
News
Apitegromab Improves Motor Function in Children With SMA
09/24/2025
Jolynn Tumolo
Treatment with the fully human monoclonal antibody apitegromab was associated with statistically significant improvements in motor function scores compared with placebo in patients with type 2 or type 3 spinal muscular atrophy (SMA).
Treatment with the fully human monoclonal antibody apitegromab was associated with statistically significant improvements in motor function scores compared with placebo in patients with type 2 or type 3 spinal muscular atrophy (SMA).
Treatment with the fully human...
09/24/2025
Neurology
A teddy bear against a blue background wearing a mask and stethoscope.
News
Phase 2 Trial Finds Risdiplam Effective and Safe in Newborns With SMA
08/21/2025
Jolynn Tumolo
Infants with presymptomatic spinal muscular atrophy (SMA) treated with risdiplam as early as 16 days of age had better functional and survival outcomes at 12 and 24 months compared with untreated infants with SMA.
Infants with presymptomatic spinal muscular atrophy (SMA) treated with risdiplam as early as 16 days of age had better functional and survival outcomes at 12 and 24 months compared with untreated infants with SMA.
Infants with presymptomatic...
08/21/2025
Neurology
News
Parkinson Disease Risk Elevated in Patients With Upper GI Mucosal Damage
09/30/2024
Jolynn Tumolo
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
Jolynn Tumolo
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
Jolynn Tumolo
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
Evi Arthur
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
An orange prescription container with generic pills spilling out in front of it
News
Antidepressant Use Not Linked to Worse Traumatic Brain Injury Outcomes
02/03/2026
Brionna Mendoza
A large nationwide cohort study found no evidence that preinjury antidepressant use increases short-term mortality, need for emergency brain surgery, or length of hospitalization following TBI.
A large nationwide cohort study found no evidence that preinjury antidepressant use increases short-term mortality, need for emergency brain surgery, or length of hospitalization following TBI.
A large nationwide cohort study...
02/03/2026
Psych Congress Network
a mother and daughter sit at a table
News
Fremanezumab Reduces Migraine Days in Pediatric Episodic Migraine
01/30/2026
Maria Mantas
In a recent randomized, placebo-controlled trial, researchers found that fremanezumab significantly reduced monthly migraine days compared with placebo in children and adolescents with episodic migraine. 
In a recent randomized, placebo-controlled trial, researchers found that fremanezumab significantly reduced monthly migraine days compared with placebo in children and adolescents with episodic migraine. 
In a recent randomized,...
01/30/2026
Neurology
fda building sign
News
FDA Approves Copper Histidinate Treatment for Pediatric Menkes Disease
01/19/2026
Brionna Mendoza
The US Food and Drug Administration (FDA) approved a copper histidinate (Zycubo) injection as the first treatment for Menkes disease in pediatric patients.
The US Food and Drug Administration (FDA) approved a copper histidinate (Zycubo) injection as the first treatment for Menkes disease in pediatric patients.
The US Food and Drug...
01/19/2026
Neurology
brain scans
News
Endovascular Thrombectomy Shows Durable 3-Year Benefit in Basilar Artery Occlusion
01/06/2026
Maria Mantas
Endovascular thrombectomy (EVT) provides durable functional and survival benefits for patients with acute basilar artery occlusion, according to long-term follow-up data from a randomized clinical trial. 
Endovascular thrombectomy (EVT) provides durable functional and survival benefits for patients with acute basilar artery occlusion, according to long-term follow-up data from a randomized clinical trial. 
Endovascular thrombectomy (EVT)...
01/06/2026
Neurology
A doctor prescribing medication for a pregnant woman
News
Antiseizure Medication Dose Adjustments During Pregnancy: Findings from the MONEAD Study
01/06/2026
Sarah Fleming
A new analysis from the US-based Maternal Outcomes and Neurodevelopmental Effects of Antiepileptic Drugs (MONEAD) study, published in Neurology, examines how antiseizure medications (ASMs) were adjusted during pregnancy and early postpartum...
A new analysis from the US-based Maternal Outcomes and Neurodevelopmental Effects of Antiepileptic Drugs (MONEAD) study, published in Neurology, examines how antiseizure medications (ASMs) were adjusted during pregnancy and early postpartum...
A new analysis from the US-based...
01/06/2026
Neurology
A doctor fills out a form while meeting with a patient
News
AAN Issues Evidence-Based Guideline for Management of Functional Seizures
12/18/2025
Sarah Fleming
A new clinical practice guideline from the American Academy of Neurology (AAN), published in Neurology, provides comprehensive recommendations for diagnosing and managing functional seizures.
A new clinical practice guideline from the American Academy of Neurology (AAN), published in Neurology, provides comprehensive recommendations for diagnosing and managing functional seizures.
A new clinical practice...
12/18/2025
Neurology
A doctor reviews test results with an older female patient
News
Late-Window IV Thrombolysis Before Transfer May Improve Stroke Outcomes
12/17/2025
Sarah Fleming
A multicenter study suggests that extended-window IV thrombolysis before thrombectomy transfer may enhance outcomes and recanalization in AIS-LVO without increasing hemorrhagic risk.
A multicenter study suggests that extended-window IV thrombolysis before thrombectomy transfer may enhance outcomes and recanalization in AIS-LVO without increasing hemorrhagic risk.
A multicenter study suggests...
12/17/2025
Neurology
News
Fine Particulate Air Pollution Linked to Greater Alzheimer Neuropathology, Study Finds
12/16/2025
Grace Taylor, MS, MA
Research published in JAMA Neurology links higher exposure to fine particulate air pollution (PM2.5) with more severe Alzheimer-related brain changes and faster cognitive decline in older adults.
Research published in JAMA Neurology links higher exposure to fine particulate air pollution (PM2.5) with more severe Alzheimer-related brain changes and faster cognitive decline in older adults.
Research published in JAMA...
12/16/2025
Annals of Long-Term Care
a man lies in bed sleeping while wearing a CPAP machine
News
Untreated Sleep Apnea Nearly Doubles Parkinson Disease Risk
12/09/2025
Brionna Mendoza
Obstructive sleep apnea (OSA) appears to nearly double the risk for later development of Parkinson disease (PD), but early continuous positive airway pressure (CPAP) treatment may attenuate that risk by 30%.
Obstructive sleep apnea (OSA) appears to nearly double the risk for later development of Parkinson disease (PD), but early continuous positive airway pressure (CPAP) treatment may attenuate that risk by 30%.
Obstructive sleep apnea (OSA)...
12/09/2025
Neurology
Two doctors review research data on a computer in a lab
News
Deramiocel Meets Key Endpoints in Phase 3 HOPE-3 Duchenne Trial
12/05/2025
Brionna Mendoza
Deramiocel, an investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD), met its primary and secondary endpoints in the phase 3 HOPE-3 trial, according to Capricor Therapeutics.
Deramiocel, an investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD), met its primary and secondary endpoints in the phase 3 HOPE-3 trial, according to Capricor Therapeutics.
Deramiocel, an investigational...
12/05/2025
Neurology

Alzheimer's Disease CME Series

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

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