A World-Class pediatric Duchenne Muscular Dystrophy program

When you first come to the Duchenne Muscular Dystrophy (DMD) program, your child will receive a whole-body assessment that will include physical tests, blood tests and muscle biopsies.

Children’s Health experts understand the specific ways that muscular dystrophy affects breathing, heart function and other aspects of your child’s health.

The most common programs and clinics our patients receive care from include:

• Programa de fallas del corazón
• Terapia ocupacional pediátrica
• Gastroenterología (GI) pediátrica
• Neurología pediátrica
• Terapia física pediátrica
• Medicina pulmonar pediátrica
• Trabajo social

También buscamos activamente nuevas y mejores formas de tratar la DMD a través de la investigación y los ensayos clínicos. La investigación continua incluye estudios para técnicas avanzadas, como la terapia génica y la tecnología de omisión de exones, que apunta a restaurar una proteína que no funciona correctamente en pacientes con DMD.

Atención conveniente y coordinada de la distrofia muscular

Generalmente, la atención de la distrofia muscular requiere que los pacientes tengan que esforzarse para coordinar varias citas en diferentes consultorios médicos. Esto puede implicar recorrer grandes distancias y faltar varios días a la escuela.

En nuestra clínica, programamos citas para que su hijo consulte a todos los médicos en una sola visita. Esta cita multidisciplinaria es más conveniente para usted y su hijo. También permite a nuestro equipo trabajar en conjunto y avanzar rápidamente con la atención que su hijo necesita.

Living with Pediatric Duchenne Muscular Dystrophy

Children with duchenne muscular dystrophy will experience muscle weakness throughout life. Eventually their muscles will start to break down causing them to need progressively more acute care and support.

DMD patients develop enlarged calf muscles, while their thigh muscles become thin. Their tongue and forearm muscles may also become enlarged. Progressive joint stiffening will reduce their flexibility and limit their ability to move independently.

Many children will develop severe spinal curvature (scoliosis).

Pulmonary complications are also common due to weakness in chest and throat muscles. When this happens, children have difficulty coughing and swallowing, which can cause frequent lung infections. Eventually, the heart muscles will begin to break down, causing cardiomyopathy.

Apoyo para el niño y la familia

Tomamos medidas adicionales para ayudar a los niños a vivir bien con un diagnóstico de DMD; y apoyamos a las familias a lo largo del camino de la atención. Nos tomamos el tiempo para informar a las familias sobre la enfermedad y cómo esta afección puede incidir en sus vidas. Estamos disponibles para ayudar con todos los aspectos de la vida con DMD, desde trabajar con las escuelas hasta conseguir sillas de ruedas y realizar otros cambios para lograr la accesibilidad.

We also aim to have each child to see themselves as capable as every other child. Our team will integrate your child’s wants and needs into their treatment and use those goals as motivation to pursue new milestones. We have a number of resources to participate in adaptive sports and other activities.

Coordinated care for complex needs

Some children may eventually need surgery for scoliosis, cataracts or heart problems. Your child’s DMD team will work closely with the inpatient care team to plan their recovery and new therapeutic needs.

All DMD patients will eventually need extensive heart and lung care. A cough-assist machine will help your child get a deeper breath and clear their airway. Many Duchenne patients eventually need a machine to help with breathing (bilevel positive airway pressure, Bi-PAP). Medicines can help the heart move blood around the body and some will need a pacemaker.

Partnering with other clinics

Some families find regular outpatient care close to their home or school. We can partner with specialists in your area and can continue to provide specialty care as needed.