source: Neuromuscular Disorders
In the last decade a number of therapeutic approaches for Duchenne muscular dystrophy has become available and there has been a proliferation of clinical trials. Several initial approaches have focused on attempts to restore dystrophin production by targeting specific groups of mutations. These have focused out of frame deletions with antisense oligonucleotides, or nonsense mutations with drugs allowing partial read through.
These attempts have led to the first drugs to be commercially available but have also raised a number of questions on trial design and on the need to further explore aspects related to inclusion criteria, duration of the study and outcome measures used. Other approaches have targeted different aspects of the pathophysiology of Duchenne. As an alternative to dystrophin restoration, another approach focuses on the use of small molecules aiming to upregulate the production of a dystrophin like protein, utrophin.
Catholic University, Italy
10.1016/j.nmd.2018.06.235 read more
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