Updates on the Evolving Role of Multidisciplinary Care Teams for the Management of Patients with Duchenne Muscular Dystrophy
Evolving Treatment and Multidisciplinary Care in Managing Duchenne Muscular Dystrophy
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A 4-year-old White child is diagnosed with Duchenne muscular dystrophy and receives initial corticosteroids
and referral to other caregivers (eg, pulmonary, cardiac, orthopedic, etc). One year later, at 5 years of age, the
parents ask about gene therapy. Is this child a candidate for gene therapy at this time?
No, the patient is too young
No, the patient is too old
No, because the patient has already received corticosteroids
Yes, the current indication for gene therapy in DMD is 5-6 years of age
Which of the following is true regarding multidisciplinary care for patients with Duchenne muscular
dystrophy?
The responsibility of leading the care team and coordinating treatment for DMD should rest almost exclusively on primary care clinicians
While PCPs should exercise shared decision making with patients, specialists should dictate to patients what treatments they should take
The care of patients with DMD could involve primary care, neurology, orthopedics, pulmonology, cardiology and several others
Generally, in a patient with DMD, clinicians beyond neurology and primary care are not necessary
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