Clinical development of novel therapies for Duchenne muscular dystrophy—Current and future

Duchenne muscular dystrophy (DMD), the most common muscle degenerative disease, is an X-linked genetic disorder caused by loss or reduction of dystrophin protein, resulting in progressive wasting, and involving skeletal, cardiac, respiratory muscles. There currently no cure for DMD, anti-inflammatory steroid conventional treatment to delay disease progression. Recently, several therapeutic approaches have been developed improve patient quality life even treat underlying cause disease. These include exon-skipping, stop-codon read-through, vector-mediated gene therapy, stem cell transplantation. Exon-skipping one promising techniques, four exon-skipping drugs received approval, including NS-065/NCNP-01 (viltolarsen) Japan USA. The read-through drug, Ataluren, has approval EU. Vector-mediated therapy transplantation are also attractive approaches, currently, clinical trials ongoing some drugs. Furthermore, studies innovative DMD treatment, such as multiple exon skipping, editing using CRISPR/Cas9 system, mesenchymal stromal (MSC)- inducible pluripotent (iPSC)-based In this review, we summarize current treatment.