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June 9, 2017Journal of Neuromuscular Diseases

Analysis of Azithromycin Monohydrate as a Single or a Combinatorial Therapy in a Mouse Model of Severe Spinal Muscular Atrophy

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Key result

Co-administration of azithromycin and an antisense oligonucleotide significantly extended survival and improved body weight and movement in a severe mouse model of spinal muscular atrophy.

Population

Severe mouse model of Spinal Muscular Atrophy (SMA)

Comparison

Azithromycin administered via ICV, alone or in… vs Appropriate controls

Design

Preclinical

Authors

EOErkan Y. OsmanMissouri CollegeCWCharles W. WashingtonGeorge Washington UniversityMSMadeline E. SimonUniversity of Missouri Hospital

Discussion

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Implication

Should not change SMA management; leaves open translation of AZM-ASO synergy to clinical trials.

Structured PICO

P
Population
Severe mouse model of spinal muscular atrophy.
I
Intervention
Azithromycin (AZM) administered via ICV, alone or in combination with an antisense oligonucleotide (ASO)
C
Comparator
Appropriate controls (AZM alone, ASO alone, or untreated)
O
Outcome
SMN protein levels in disease-relevant tissues and phenotypic parameters (survival, body weight, movement)surrogate

Azithromycin combined with an antisense oligonucleotide improves survival and phenotype in a severe mouse model of SMA.

Cite This Study

Osman et al. (2017) studied Spinal muscular atrophy (SMA). Azithromycin (AZM) alone or in combination with an antisense oligonucleotide (ASO) vs. Controls, AZM alone, or ASO alone was evaluated on SMN protein levels, survival, body weight, and movement. Co-administration of azithromycin and an antisense oligonucleotide significantly extended survival and improved body weight and movement in a severe mouse model of spinal muscular atrophy.

synapsesocial.com/papers/6a9901c9aa63da60ef78c9a0https://doi.org/10.3233/jnd-170230
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Also Consider

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  1. 1Eukaryotic release factors (eRFs) history2003 · 122 citations
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  3. 3Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular Atrophy2011 · 66 citations
  4. 4Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy2011 · 570 citations
  5. 5Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy2013 · 88 citations