Clinical Unmet Need in Duchenne Muscular Dystrophy (DMD)

DMD Is a Rapidly Progressing, Severely Debilitating, Rare Disease of Muscle Wasting1

Signs and Symptoms1,2
Muscle damage;
inflammation; loss
of muscle strength
and function
Progressive heart
dysfunction,
cardiomyopathy,
heart failure
Weakened
diaphragm leading
to need for assisted
ventilation
Low bone
mineral density
Learning and
cognitive
difficulties

Treatment Recommendations Include Corticosteroids as Part of the Standard of Care in DMD3

  • Corticosteroids slow the decline in muscle strength and function in DMD, in part due to effects on inflammation1,3
  • Guidelines recommend initiating corticosteroids before substantial physical decline3

Chronic Corticosteroid Use Is Associated With Treatment-Limiting Adverse Effects3,4

Adverse effects are the primary reason (in up to 65% of patients) for discontinuation of corticosteroid treatment5

Worry about side effects was the #1 reason (25%) for not initiating corticosteroid treatment5

Data from the Duchenne Registry including 1627 responses from individuals with DMD aged ≤35 years from the United States.

Potentially Treatment-Limiting Adverse Effects*

  • Changes in behavior, growth delay, and decline in bone health are important concerns related to chronic corticosteroid use3,6-9
  • Additional treatment-limiting adverse effects may include cushingoid appearance, weight gain, hirsutism, and cataracts4,7
  • These are not all the possible adverse effects of corticosteroid treatment
Behavior changes6†
May necessitate modifications and disruptions in treatment3,4,7
Growth delay7‡
Negatively affects psychosocial well-being and adherence to therapy9
Changes to bone mineral density7‡
Osteoporosis from corticosteroids increases risk8
Bone fractures7‡
Substantial risk factor for premature, permanent loss of ambulation8
May lead to chronic back pain and spine deformity8
❮ ❯

†Based on data from 277 patients (mean age, 10.9 years) treated with corticosteroids from the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG-DNHS). Patients were considered treated with corticosteroids if they were taking corticosteroids for ≥1 year.6

‡Based on data from 97 patients with DMD aged 10 to <16 years from the Comprehensive Neuromuscular Center (CNC) at Cincinnati Children’s Hospital Medical Center treated with corticosteroids for ≥3 years.7

Why
AGAMREE®?

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Proven Efficacy
and Safety

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