Clinical Unmet Need in Duchenne Muscular Dystrophy (DMD)

Treatment Recommendations Include Corticosteroids as Part of the Standard of Care in DMD1

  • Corticosteroids slow the decline in muscle strength and function in DMD, in part due to effects on inflammation1,2
  • Guidelines recommend initiating corticosteroids before substantial physical decline1

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

Among ambulatory patients with DMD who have been treated with corticosteroids for >1 year4:

37%

Growth failure delay

37%

Behavioral changes

22%

Bone changes or fractures

55%

Cushingoid appearance

65%

Weight gain

6%

Excessive hair growth

15%

Cataract

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

*Data from the Duchenne Registry including 1627 responses from individuals with DMD aged ≤35 years from the United States.5

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

Potentially Treatment-Limiting Adverse Effects*

  • Changes in behavior, growth delay, and decline in bone health are important concerns related to chronic corticosteroid use1,4,6-8
  • Additional treatment-limiting adverse effects may include cushingoid appearance, weight gain, hirsutism, and cataracts3-6
37%

Behavior changes4†

May necessitate modifications and disruptions in treatment1,3,6

Up to
72%

Growth delay6‡

Negatively affects psychosocial well-being and adherence to therapy8

>70%

Changes to bone mineral density6‡

Osteoporosis from corticosteroids increases risk7

>75%

Bone fractures6‡

Substantial risk factor for premature, permanent loss of ambulation7

May lead to chronic back pain and spine deformity7

  • Changes in behavior, growth delay, and decline in bone health are important concerns related to chronic corticosteroid use1,4,6-8
  • Additional treatment-limiting adverse effects may include cushingoid appearance, weight gain, hirsutism, and cataracts3-6

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.4

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.6

Why
AGAMREE®?

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Proven Efficacy
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