Neurology

Duchenne Muscular Dystrophy Program

image of child

Muscular dystrophy refers to a group of about 30 genetic conditions that cause muscle weakness and loss of muscle mass. These conditions affect the muscles that control movement. They make it hard to do everyday tasks such as walking, climbing stairs, or lifting objects. Over time, muscular dystrophy can also involve other systems in the body. Depending on the type, it may affect the heart, lungs, gastrointestinal system, skeletal alignment, and even learning and behavior. Some patients need comprehensive, long-term care.

Duchenne muscular dystrophy is the most common form of muscular dystrophy. Symptoms usually appear in boys between ages two and four. Early signs include frequent falls, difficulty running or climbing, trouble getting up from the floor, and delayed motor milestones. As the condition progresses, mobility becomes increasingly limited, and many individuals eventually require a wheelchair. Duchenne can also affect breathing, heart function, and other body systems. While there is no cure, current medical therapies can slow disease progression, manage symptoms, and improve quality of life.

At Mount Sinai, our highly skilled and compassionate specialists can help. We provide advanced diagnostic and treatment options and comprehensive care. Our team combines the latest research with individualized treatment plans, taking the time to understand your family’s needs.

Specialist Care

Duchenne/Becker Muscular Dystrophy care is complex. It requires the expertise of many specialists. At Mount Sinai, muscular dystrophy care begins in our Neuromuscular Program. This comprehensive care clinic provides access to several specialists at one location, in a single visit. It also streamlines appointments, improves provider communication, and helps us address every aspect of your child’s health.

Your child’s care team may include specialists in:

  • Cardiology
  • Dentistry
  • Endocrinology
  • Neurology
  • Nutrition
  • Orthopedic surgery
  • Palliative care
  • Physical medicine and rehabilitation
  • Pulmonology
  • Urology

In addition, we can refer your family to specialists in:

  • Developmental pediatrics
  • Pediatric general surgery
  • Speech and feeding therapy
  • Physical therapy
  • Occupational therapy
  • Wheelchair and mobility services

Our physicians are also experienced in coordinating care with outside providers, ensuring a smooth transition if your child is coming to us from another institution or currently being treated elsewhere.

Services We Offer

We provide compassionate, comprehensive care supported by the latest research and medical technology. We tailor your child’s treatment plan to meet their needs. Our evidence-based tools and approaches include:

  • Early treatment of heart issues
  • Echocardiograms to check heart function
  • Genetic testing and counseling
  • High-technology biomarkers, including magnetic resonance imaging
  • Lung function testing
  • Sleep study (non-invasive treatment to restore circadian rhythms)

We work with researchers at the Icahn School of Medicine at Mount Sinai to explore new ways to improve care for children with muscular dystrophy. Our patients may be able to participate in clinical trials. As we grow, expanding the breath of clinical trials is a major focus for our program. This can offer access to innovative diagnostic methods and treatments that are not yet widely available.

Transition to Adult Care

With advances in treatment options, children with muscular dystrophy are living longer, fuller lives. As they reach their early teens, we begin preparing for the transition to adult care. We coordinate a smooth handoff from pediatric specialists to adult clinicians. Meanwhile, we continue to address ongoing medical needs.

We also address your child’s long-term goals. We discuss plans for education, employment, and independent living. We work closely with community providers, such as social workers and vocational programs. In this way, we help your child build toward long-term success and quality of life.

Meet Our Team

Our team of health care specialists works together to meet all the needs of your child. We have special expertise in muscular dystrophy.

Clinical Director of Duchenne Muscular Dystrophy Program
Bruce D. Gelb, MD
Cardiology

Director of Duchenne Muscular Dystrophy Program
Carol C. Gregorio, PhD
Medicine/Cardiology

Director of Comprehensive Care Neuromuscular Program
Sheena Ranade, MD
Orthopedic Surgery

Program Manager
Jennifer Gomez, CPNP

Clinical Care Team
Deepak Bhatt, MD, MPH, MBA
Cardiology

Anuradha Lala-Trindade, MD
Cardiology

Jacqueline Lamour, MD
Cardiology

Peter S. Midulla, MD
General Surgery

Kathryn Scharbach, MD
Palliative Care, Complex Care Pediatrics

David Cancel, MD
Physical Medicine and Rehabilitation

Jason Bronstein, MD
Pulmonology, Sleep Medicine

Oren Cohen, MD
Pulmonology, Sleep Medicine

Monica Kraft, MD 
Pulmonology

Neha Malhotra, MD
Urology

Director, Pediatric Neurology
Walter J. Molofsky, MD