What is the clinical evidence from this study?

Study design: Other. Population: Nemaline myopathy. Intervention: L-tyrosine, L-carnitine, taurine, and creatine vs. Water (vehicle control). Primary outcome: Skeletal muscle function (distance and speed travelled) and phenotypic severity at 6 dpf (p=ns).

What question did this study set out to answer?

To evaluate whether common self-administered dietary supplements can improve skeletal muscle function and pathology in a zebrafish model of nebulin-related nemaline myopathy.

May 30, 2018Acta Neuropathologica CommunicationsOpen Access

Testing of therapies in a novel nebulin nemaline myopathy model demonstrate a lack of efficacy

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Key result

Treatment with L-tyrosine, L-carnitine, taurine, or creatine did not significantly improve skeletal muscle function or pathology in a zebrafish model of nebulin nemaline myopathy.

Why the study?

Do L-tyrosine, L-carnitine, taurine, or creatine improve skeletal muscle function in a zebrafish model of nebulin nemaline myopathy?

Population

Zebrafish model for nemaline myopathy caused by a mutation in nebulin (neb-/- mutants, sa906 strain)

Comparison

L-tyrosine, L-carnitine, taurine, or creatine… vs Water (vehicle) added to embryo medium

Design

Preclinical, Randomization of both the position of the fish within the plates…

Follow-up

6 days post fertilization

Authors

TSTamar E. SztalAustralian Regenerative Medicine Institute EMEmily A. McKaigeUniversity of Copenhagen CWCaitlin WilliamsCornell University

Discussion

Member takes

Implication

These supplements showed no benefit in a zebrafish nemaline myopathy model; leaves open efficacy in patients and need for further studies.

Key Points

To evaluate whether common self-administered dietary supplements can improve skeletal muscle function and pathology in a zebrafish model of nebulin-related nemaline myopathy.
Characterized a novel zebrafish model carrying a mutation in nebulin that exhibits electron-dense nemaline bodies and reduced muscle function.
Treated the zebrafish model with four individual supplements: L-tyrosine, L-carnitine, taurine, or creatine.
Assessed muscle pathology and locomotor performance after supplement administration.
Zebrafish with the nebulin mutation successfully replicated patient phenotypes, including characteristic nemaline bodies and impaired locomotion.
Treatment with L-tyrosine, L-carnitine, taurine, or creatine produced no significant improvement in skeletal muscle pathology or locomotor function.

Structured PICO

Do L-tyrosine, L-carnitine, taurine, or creatine improve skeletal muscle function in a zebrafish model of nebulin nemaline myopathy?

Population

Zebrafish neb-/- mutants modeling nemaline myopathy treated with various supplements from 28 hours post fertilization to 6 days post fertilization.

Intervention

L-tyrosine (10 μM), L-carnitine (10 mM), taurine (1 mM), or creatine (100 μM) added to embryo medium from 28 hours post fertilization until 6 days post fertilization

Comparator

Water (vehicle) added to embryo medium

Outcome

Skeletal muscle function (distance and speed travelled) and phenotypic severity of skeletal muscle at 6 days post fertilizationsurrogate

Main Result

p-value: p=ns

Commonly self-administered supplements (L-tyrosine, L-carnitine, taurine, creatine) failed to improve skeletal muscle function in a zebrafish model of nemaline myopathy.

Limitations

Animal model may not fully reflect human disease
Facial musculature could not be assessed
Only specific maximum non-toxic doses were tested

Cite This Study

Sztal et al. (2018) studied Nemaline myopathy. L-tyrosine, L-carnitine, taurine, and creatine vs. Water (vehicle control) was evaluated on Skeletal muscle function (distance and speed travelled) and phenotypic severity at 6 dpf (p=ns). Treatment with L-tyrosine, L-carnitine, taurine, or creatine did not significantly improve skeletal muscle function or pathology in a zebrafish model of nebulin nemaline myopathy.

synapsesocial.com/papers/6a9846db329299b5dce34e1b https://doi.org/10.1186/s40478-018-0546-9

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May 30, 2018Acta Neuropathologica CommunicationsOpen Access

Testing of therapies in a novel nebulin nemaline myopathy model demonstrate a lack of efficacy

View Full Paper

Ask AI

Bookmark

Key result

Treatment with L-tyrosine, L-carnitine, taurine, or creatine did not significantly improve skeletal muscle function or pathology in a zebrafish model of nebulin nemaline myopathy.

Why the study?

Do L-tyrosine, L-carnitine, taurine, or creatine improve skeletal muscle function in a zebrafish model of nebulin nemaline myopathy?

Population

Zebrafish model for nemaline myopathy caused by a mutation in nebulin (neb-/- mutants, sa906 strain)

Comparison

L-tyrosine, L-carnitine, taurine, or creatine… vs Water (vehicle) added to embryo medium

Design

Preclinical, Randomization of both the position of the fish within the plates…

Follow-up

6 days post fertilization

Authors

TSTamar E. SztalAustralian Regenerative Medicine Institute EMEmily A. McKaigeUniversity of Copenhagen CWCaitlin WilliamsCornell University

Discussion

Member takes

Implication

These supplements showed no benefit in a zebrafish nemaline myopathy model; leaves open efficacy in patients and need for further studies.

Key Points

To evaluate whether common self-administered dietary supplements can improve skeletal muscle function and pathology in a zebrafish model of nebulin-related nemaline myopathy.
Characterized a novel zebrafish model carrying a mutation in nebulin that exhibits electron-dense nemaline bodies and reduced muscle function.
Treated the zebrafish model with four individual supplements: L-tyrosine, L-carnitine, taurine, or creatine.
Assessed muscle pathology and locomotor performance after supplement administration.
Zebrafish with the nebulin mutation successfully replicated patient phenotypes, including characteristic nemaline bodies and impaired locomotion.
Treatment with L-tyrosine, L-carnitine, taurine, or creatine produced no significant improvement in skeletal muscle pathology or locomotor function.

Structured PICO

Do L-tyrosine, L-carnitine, taurine, or creatine improve skeletal muscle function in a zebrafish model of nebulin nemaline myopathy?

Population

Zebrafish neb-/- mutants modeling nemaline myopathy treated with various supplements from 28 hours post fertilization to 6 days post fertilization.

Intervention

L-tyrosine (10 μM), L-carnitine (10 mM), taurine (1 mM), or creatine (100 μM) added to embryo medium from 28 hours post fertilization until 6 days post fertilization

Comparator

Water (vehicle) added to embryo medium

Outcome

Skeletal muscle function (distance and speed travelled) and phenotypic severity of skeletal muscle at 6 days post fertilizationsurrogate

Main Result

p-value: p=ns

Commonly self-administered supplements (L-tyrosine, L-carnitine, taurine, creatine) failed to improve skeletal muscle function in a zebrafish model of nemaline myopathy.

Limitations

Animal model may not fully reflect human disease
Facial musculature could not be assessed
Only specific maximum non-toxic doses were tested

Cite This Study

synapsesocial.com/papers/6a9846db329299b5dce34e1b https://doi.org/10.1186/s40478-018-0546-9

Also Consider

Synapse has enriched 4 closely related papers on similar clinical questions. Consider them for comparative context:

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