John F. Brandsema, MD

Duchenne trials accelerate with next-gen gene therapy, muscle-protecting drugs, and improved exon-skipping approaches—aiming for safer, longer-lasting benefits and easier dosing.

Clinicians tailor Duchenne treatment over time, balancing steroid benefits, side effects, ambulation changes, and required monitoring for newer therapies and families.

Coordinated Duchenne care transitions align cardiology, pulmonary and neuromuscular handoffs, protecting families from risky gaps after 18.

Clinicians tailor Duchenne steroid therapy over time—balancing benefits, side effects, and family capacity for monitoring newer treatments like gene therapy and givinostat.

See how care coordinators sync cardiology, pulmonology and neuromuscular visits so Duchenne teens move to adult care without gaps.

Clinicians weigh vamorolone vs prednisone/deflazacort, tailoring growth and weight goals while managing tricky steroid switches and adrenal risks.

Clinicians weigh vamorolone vs prednisone/deflazacort for young boys, balancing growth, weight, and adrenal safety during steroid transitions.

Duchenne care now reaches adulthood—discover when to plan transition, boost teen independence, and find adult clinics that support lifelong needs.

Explore today’s Duchenne muscular dystrophy treatment options, from corticosteroids and givinostat to gene therapy and exon skipping, plus evolving dosing debates.

Clinicians weigh safety, dosing, and combo therapies in Duchenne, balancing infusion burden, ethics, and gene-transfer risks before decline.

Explore today’s DMD treatment toolkit—from steroids and givinostat to gene therapy and exon skipping—plus dosing debates shaping care.

A teen with Duchenne shows how exon-skipping and steroids shape care, while clinicians stress realistic expectations amid varied progression.

Learn how DMD clinics coordinate neurology, cardio, pulm, rehab and mental health—without overwhelming visits—using nurse coordinators and team huddles.

Clinicians detail modern Duchenne care: essential multidisciplinary teams, access workarounds, and family-centered visits as therapies accelerate.

The child neurologist at Children’s Hospital of Philadelphia discussed the real-world safety data of givinostat use in patients with Duchenne muscular dystrophy at the 2026 MDA Conference. [WATCH TIME: 5 minutes]

Experts discuss Duchenne muscular dystrophy's pathophysiology, disease progression, and innovative treatment options to enhance patient care and outcomes.

Experts discuss Duchenne muscular dystrophy's pathophysiology, disease progression, and innovative treatment options to enhance patient care and outcomes.

An expert discusses how older Duchenne muscular dystrophy patients in advanced stages face uncertainty regarding newer therapies since most research focuses on younger children, but gene therapy and other treatments remain considerations alongside comprehensive specialty care for managing complex comorbidities.

An expert discusses how proactive cardiac care should begin at diagnosis with regular monitoring, while pulmonary function typically peaks around age 8 to 10 before declining, requiring early intervention with breathing exercises and assistive devices before problems become severe.

An expert discusses how a 16-year-old postambulatory DMD patient exemplifies typical challenges including social withdrawal and increasing care needs, emphasizing the critical importance of comprehensive interdisciplinary care teams despite families' perceptions of limited treatment options.

An expert discusses how treatment for the 13-year-old patient should optimize standard care with cardiac and pulmonary monitoring while considering newer therapies like givinostat and casimersen, plus exploring research trials for gene transfer and muscle health approaches.

An expert discusses how genetic therapies like exon skipping and gene transfer aim to restore dystrophin function but face challenges in delivering complete protein restoration and achieving equivalent effectiveness across skeletal, cardiac, and pulmonary muscles.

An expert discusses how a 13-year-old boy with Duchenne muscular dystrophy faces the challenging transition to complete loss of ambulation, emphasizing the importance of gradual introduction of assistive devices and psychological support during this developmentally difficult period.

An expert discusses how a 13-year-old boy with Duchenne muscular dystrophy faces the challenging transition to complete loss of ambulation, emphasizing the importance of gradual introduction of assistive devices and psychological support during this developmentally difficult period.

The pediatric neurologist at Children’s Hospital of Philadelphia gave perspective on some of the main issues the clinical community is figuring out with gene therapies and their integration to clinical practice. [WATCH TIME: 3 minutes]

The pediatric neurologist at Children’s Hospital of Philadelphia provided background on a notable track session at the upcoming MDA Clinical & Scientific Conference covering updates in gene therapy for neuromuscular conditions. [WATCH TIME: 4 minutes]

Experts discuss the future of Duchenne care, emphasizing proactive management, multidisciplinary collaboration, and the need for adult-focused treatment strategies.

Experts discuss the evolving landscape of Duchenne muscular dystrophy management, emphasizing the need for effective therapies and improved quality of life.

Discover effective strategies for optimizing steroid regimens in patients, balancing benefits and side effects for tailored treatment plans.

Explore the essential multidisciplinary approach to managing Duchenne muscular dystrophy, highlighting key specialties and best practices for optimal patient care.