Jornal de Ciências Farmacêuticas e Medicamentos Emergentes

Eteplirsen: Unattainable Attained

Mukta Sharma*, Naina Goel, Ashwin K Jainarayanan

Rare diseases are highly diverse and it is critical to know the signs that predict the development, progression and manifestation of disease. Though a very small number of people get affected by the rare diseases but its impact is outrageous, Duchenne Muscular Dystrophy (DMD) being one such rare genetic disorder is characterized by progressive muscle deterioration and wasting. An overall estimate of prevalence of DMD reflects 30 per 100,000 populations (1 in 3000 or~0.029%).