… Antisense therapy using chemically engineered antisense oligonucleotides (ASOs) … disorders
like Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) [1, 2]. ASOs …

… We explore how genetic modifiers could be used to predict cardiorespiratory natural history
and how manipulation of such modifiers might represent a promising therapeutic strategy. …

… gene SMN2 (6, 7). Loss of α motor neurons and muscle atrophy cause severe muscle
weakness, particularly of the proximal limb, truncal, and bulbar muscles (8). Patients show a wide …

… overview of the advanced gene therapies for motor neuron diseases (ie, … muscular atrophy)
and muscular dystrophies (ie, Duchenne muscular dystrophy, limb-girdle muscular dystrophy…

… Facioscapulohumeral muscular dystrophy (FSHD is a hereditary skeletal muscle disorder
that … latter case, DUX4 de-repression is caused by a malfunction of D4Z4 chromatin modifiers. …

… Patients not using GC therapy and carrying “distal” DMD mutations tended to lose mobility …
DMD gene. We did not show statistically significant effects of the selected genetic modifiers on …

… measures and opportunities for therapy based in the actual landscape of new treatments.
This review discusses the challenges and new perspectives of genetic counseling in SMA. …

… atrophy and respiratory failure due to diaphragmatic palsy [35, 37]. It is also known as distal
spinal muscular atrophy 1 (DMSA1) and distal hereditary motor … of the genetic modifier on …

… To dissect this, we turn to genetic modifiers of SMA. Understanding why these modifiers
suppress … Taken together, our results demonstrate that plastin is a conserved genetic modifier of …

… Duchenne muscular dystrophy (DMD) is a relatively widespread genetic disease which
develops as a result of a mutation in the gene DMD encoding dystrophin. In this review, animal …