Duchenne Muscular Dystrophy (DMD) Pipeline Drugs Assessment: Clinical Trails Analysis, Player Profiles, Collaborations, Key Targets, Geographic Focus, and Data Publications, 2019 - 2030

Duchenne muscular dystrophy is a genetic disorder which causes progressive muscle degeneration and weakness, and it is one of the nine types of muscular dystrophy. Duchenne muscular dystrophy mostly affects males, but in rare cases occurs in females. Onset of the disease is generally 3 to 4 years of age and begins on hips, pelvic region, shoulders, and upper legs. Symptoms may include delayed or difficulty inability to stand, walk and difficulty in speaking.

Disease is diagnosed through the patient history, blood test for creatinine phosphokinase levels, molecular genetic testing, and muscle biopsy. There is no cure available for the disease till now. But the medications are available forrelieving the symptoms of the disease. Eteplirsen is used to treat the patients with specific gene mutation of gene that leads to DMD. Deflazacort, approved in 2017, becoming first corticosteroid to treat Duchenne muscular dystrophy.

 Key Developments:

• In November 2016, Wave Life Sciences Ltd. Initiated the Phase I clinical trial for WVE-210201 Duchenne muscular dystrophy patients amenable to exon 51 skipping.