Eteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative Patient Experience Study
J Iff, C Carmichael, S McKee, I Sehinovych… - Advances in …, 2024 - Springer
Introduction Duchenne muscular dystrophy (DMD) is characterized by rapid functional
decline. Current available treatment options aim to delay disease progression or stabilize …
decline. Current available treatment options aim to delay disease progression or stabilize …
[HTML][HTML] Bridging the Gap: Translational Medicine and Novel Therapies in Neuromuscular Diseases
M Filosto - Journal of Integrative Neuroscience, 2024 - imrpress.com
Neuromuscular diseases encompass a wide range of disorders that affect motor neurons,
peripheral nerves, and muscles. The impact of most of these conditions can be profound …
peripheral nerves, and muscles. The impact of most of these conditions can be profound …
Mathematical modeling of the progression of Duchenne Muscular Dystrophy in San Diego in an era of allele specific oligonucleotide therapy
S Kingsmore, D Baun, L Tobin, H Nguyen, M Arenchild… - 2024 - researchsquare.com
Duchenne muscular dystrophy (DMD) is a relatively common, severe, progressive, X-linked
muscle disorder caused by pathogenic variation in the dystrophin gene. Affected status can …
muscle disorder caused by pathogenic variation in the dystrophin gene. Affected status can …