Investigational FSHD Drug Did Little to Change DUX4 Expression
Losmapimod was associated with small improvements in fat infiltration, upper arm function
Source: medpagetoday.com
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Losmapimod was associated with small improvements in fat infiltration, upper arm function
The highest proportions of patients with severe ocular impairment at baseline showed greater improvements in symptom severity on ravulizumab in comparison with placebo after 26 weeks.
First patient dosed with ATA-100 gene replacement therapy for LGMD-R9 On-going multicenter, Phase 1/2 study evaluating safety, pharmacodynamic and efficacy of ATA-100 Atamyo Therapeutics