News
Media
Conferences
CME/CE
Partners
Publications
Resources
Subscribe

Opinion|Videos|July 10, 2024

Improvement in Management of Duchenne Muscular Dystrophy in Recent Years

Author(s)Emma Ciafaloni, MD

A neurologist discusses how recent approvals of therapies for Duchenne muscular dystrophy have improved patient outcomes.

Episodes in this series

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 1 Clinical Presentation and Diagnosis of Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 2 Communication of Duchenne Muscular Dystrophy Diagnosis and Coordination of Care

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 3 Current and Emerging Treatment Options and Management of Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 4 Gene Therapy for Treatment of Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 5 Targeting Pathways Downstream of Dystrophin: Vamorolone and Givinostat in Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 6 Improvement in Management of Duchenne Muscular Dystrophy in Recent Years

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 7 Multidisciplinary Care in Duchenne Muscular Dystrophy

Video content above is prompted by the following questions:

How have these recent approvals improved the management of Duchenne muscular dystrophy (DMD)?
- Share your experience with recent approvals in DMD.

Newsletter

Keep your finger on the pulse of neurology—subscribe to NeurologyLive for expert interviews, new data, and breakthrough treatment updates.

Related Content

This Week on NeurologyLive® — October 20, 2025

ByIsabella Ciccone, MPH

October 20th 2025

NeurologyLive® Brain Games: October 19, 2025

October 19th 2025

Elecsys pTau181 Test Cleared, Tividenofusp Alfa PDUFA Pushed Back, Phase 4 LOTUS Study Highlights Real-World Benefit of Trofinetide

ByNeurologyLive® Staff

October 18th 2025

Optimizing Pediatric NeuroICU Treatment Through Precision and Collaboration

October 16th 2025

Phase 2 OPAL Study to Test Muscle-Targeting Therapy Apitegromab in Infants With SMA

October 15th 2025

Latest CME

4th Women in Neurology Conference

In-Person Event

4th Women in Neurology Conference

October 24-26, 2025

SimulatED™: Understanding the Role of Genetic Testing in Patient Selection for Anti-Amyloid Therapy

Case-based Simulation

SimulatED™: Understanding the Role of Genetic Testing in Patient Selection for Anti-Amyloid Therapy

Nicholas Doher, DO; Babak Tousi, MD

Medical Crossfire®: Integrating Real-World Data to Improve Outcomes for Patients With Multiple Sclerosis

Medical Crossfire®: Integrating Real-World Data to Improve Outcomes for Patients With Multiple Sclerosis

Carrie M. Hersh, DO, MSc, FAAN; Anna Shah, MD

Medical Crossfire® - Optimizing Management for Patients With Generalized Myasthenia Gravis: Focus on Complement Inhibitors

Medical Crossfire® - Optimizing Management for Patients With Generalized Myasthenia Gravis: Focus on Complement Inhibitors

James F. Howard Jr, MD; Nicholas J. Silvestri, MD, FAAN; Neelam Goyal, MD

Burst CME™: Optimizing Migraine Management – Addressing Unmet Needs, Individualizing Care for Diverse Populations, and Utilizing CGRP Targeted Agents

Burst CME™: Optimizing Migraine Management – Addressing Unmet Needs, Individualizing Care for Diverse Populations, and Utilizing CGRP Targeted Agents

Jessica Ailani, MD, FAHS, FAAN; Paul G. Mathew, MD, DNBPAS, FAAN, FAHS

Burst CME™: Optimizing the Use of CGRP Targeted Agents for the Treatment of Migraine

Burst CME™: Optimizing the Use of CGRP Targeted Agents for the Treatment of Migraine

Jessica Ailani, MD, FAHS, FAAN; Paul G. Mathew, MD, DNBPAS, FAAN, FAHS

Burst CME™: The Patient Journey – Unmet Needs From Diagnosis Through Management of Migraine

Burst CME™: The Patient Journey – Unmet Needs From Diagnosis Through Management of Migraine

Jessica Ailani, MD, FAHS, FAAN; Paul G. Mathew, MD, DNBPAS, FAAN, FAHS

Evolving Perspectives in Alzheimer Disease : Reaching an Earlier Diagnosis, Understanding Neuroinflammation, and Exploring Therapeutic Advances

Evolving Perspectives in Alzheimer Disease : Reaching an Earlier Diagnosis, Understanding Neuroinflammation, and Exploring Therapeutic Advances

Alireza Atri, MD, PhD; Erik S. Musiek, MD, PhD; Malú Gámez Tansey, PhD

Community Practice Connections™: Optimizing the Management of Tardive Dyskinesia—Addressing the Complexity of Care With Targeted Treatment

Community Practice Connections™: Optimizing the Management of Tardive Dyskinesia—Addressing the Complexity of Care With Targeted Treatment

Ilan Melnick, MD; Alejandro Alva, MD; Linda Trinh, DNP, PMHNP, FNP, MPH

Burst CME™: Setting the Stage – Individualizing Migraine Care for Diverse Populations Across Care Settings

Burst CME™: Setting the Stage – Individualizing Migraine Care for Diverse Populations Across Care Settings

Jessica Ailani, MD, FAHS, FAAN; Paul G. Mathew, MD, DNBPAS, FAAN, FAHS

Expert Illustrations & Commentaries™: Visualizing the Role of B Cells as Therapeutic Targets for Generalized Myasthenia Gravis

Expert Illustrations & Commentaries™: Visualizing the Role of B Cells as Therapeutic Targets for Generalized Myasthenia Gravis

October 31, 2025

Cases and Conversations™: Applying Best Practices to Prevent Shingles in Your Practice

Cases and Conversations™: Applying Best Practices to Prevent Shingles in Your Practice

Paul G. Auwaerter, MD, MBA; Paul P. Doghramji, MD, FAAFP; Aruna Subramanian, MD

Unlocking the Future of Glioma Care: Integrating Recent Advances to Personalize Treatment

In-Person + Virtual Event

Unlocking the Future of Glioma Care: Integrating Recent Advances to Personalize Treatment

November 21, 2025

Patient, Provider, and Caregiver Connection™: Pediatric Myasthenia Gravis—Current Treatment and Emerging Concepts

Patient, Provider, and Caregiver Connection™: Pediatric Myasthenia Gravis—Current Treatment and Emerging Concepts

John F. Brandsema, MD; Abigail N. Schwaede, MD; Samuel J. Mackenzie, MD, PhD

Mind Moments™: Optimizing Management for Patients with Generalized Myasthenia Gravis

Mind Moments™: Optimizing Management for Patients with Generalized Myasthenia Gravis

James F. Howard Jr, MD

Cases and Conversations™: Navigating the Complexities of Managing Myasthenia Gravis in Pediatric and Pregnant Patient Populations

Cases and Conversations™: Navigating the Complexities of Managing Myasthenia Gravis in Pediatric and Pregnant Patient Populations

John Brandsema, MD; Neelam Goyal, MD

Identifying and Treating CIDP in the Modern Era

Identifying and Treating CIDP in the Modern Era

Jeffrey A. Allen, MD; Karissa L. Gable, MD

Cases and Conversations™: Little Voices, Big Challenges – Comprehensive Care for Pediatric Spinal Muscular Atrophy

Cases and Conversations™: Little Voices, Big Challenges – Comprehensive Care for Pediatric Spinal Muscular Atrophy

John Brandsema, MD; Carla D. Zingariello, DO

A Breath of Strength: Managing Cancer Associated LEMS and Lung Cancer as One

A Breath of Strength: Managing Cancer Associated LEMS and Lung Cancer as One

Estelamari Rodriguez, MD, MPH; Millie Das, MD; Ruham Nasany, MD

Striking the Right Nerve: Managing Cancer Associated LEMS in Lung Cancer Patients

Striking the Right Nerve: Managing Cancer Associated LEMS in Lung Cancer Patients

Anne Chiang, MD, PhD; J. Douglas Miles, MD, PhD, FAAN; Suma Satti, MD

Patient, Provider, and Caregiver Connection: Improving Diagnosis and Care for Adolescent Myasthenia Gravis Patients – A New Frontier

Patient, Provider, and Caregiver Connection: Improving Diagnosis and Care for Adolescent Myasthenia Gravis Patients – A New Frontier

Abigail N. Schwaede, MD; Emmanuelle (Noelle) Tiongson, MD

A New Era in NMOSD Treatment: Optimizing Therapeutic Transitions and Reducing Patient Burden

A New Era in NMOSD Treatment: Optimizing Therapeutic Transitions and Reducing Patient Burden

Shamik Bhattacharyya, MD, MS, FAAN; Mirla Avila, MD; Dean Wingerchuk, MD

PER Podcast™: Maximizing the Multidisciplinary Care Outcomes of Patients with Amyotrophic Lateral Sclerosis (ALS)

PER Podcast™: Maximizing the Multidisciplinary Care Outcomes of Patients with Amyotrophic Lateral Sclerosis (ALS)

Richard Bedlack, MD, PhD

PER Podcast™: Best Strategies for Approaching the Diagnosis of Amyotrophic Lateral Sclerosis (ALS)

PER Podcast™: Best Strategies for Approaching the Diagnosis of Amyotrophic Lateral Sclerosis (ALS)

Terry Heiman-Patterson, MD

PER Postgame™: Updates from ANNA in the Management of IgA Nephropathy

PER Postgame™: Updates from ANNA in the Management of IgA Nephropathy

Gerald Bernard Appel, MD; Ellie Kelepouris, MD, FACP, FAHA

Patient, Provider, and Caregiver Connection: Turning a New Leaf in Acute Pain Management – How Recent Advancements Impact the Treatment Paradigm

Patient, Provider, and Caregiver Connection: Turning a New Leaf in Acute Pain Management – How Recent Advancements Impact the Treatment Paradigm

Jeffrey Gudin, MD; Jeffrey Bettinger, PharmD

Trending on NeurologyLive - Clinical Neurology News and Neurology Expert Insights

Early Results Show Symptom Gains in First Patient Dosed With UX-DA001 Cell Therapy for Parkinson Disease

Bringing Biomarkers to the Bedside: The Clinical Promise of Abbott’s TBI Test

Understanding Key Clinical and Genetic Biomarkers in Huntington Disease Research: Sarah O'Shea, MD, MSc

De-Escalation Strategies in Multiple Sclerosis: Oral Therapies and Cladribine

COAST Trial: Evaluating Ozanimod Step-Down in Multiple Sclerosis

© 2025 MJH Life Sciences^®

All rights reserved.