GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE

Conditions

Pompe Disease

Brief summary

Change in glycogen concentration of the hamstring muscles between baseline and 12-month follow-up

Detailed description

Change in glycogen concentration of the calf, hamstring, anterior thigh, and lumbar muscles over time – both within the first treatment cycle, and at 3, 6 and 12-months follow-up, Change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 3, 6 and 12-months follow-up., Correlations between baseline glycogen levels and change in FVC between baseline and 12-months follow-up., Correlations between baseline glycogen levels and change in 6MWT between baseline and 12-months follow-up., Correlations between baseline glycogen levels and change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 12-months follow-up.

Related papers

No related papers found yet.

Interventions

DRUGNexviadyme 100 mg powder for concentrate for solution for infusion

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Change in glycogen concentration of the hamstring muscles between baseline and 12-month follow-up	—

Secondary

Measure	Time frame
Change in glycogen concentration of the calf, hamstring, anterior thigh, and lumbar muscles over time – both within the first treatment cycle, and at 3, 6 and 12-months follow-up, Change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 3, 6 and 12-months follow-up., Correlations between baseline glycogen levels and change in FVC between baseline and 12-months follow-up., Correlations between baseline glycogen levels and change in 6MWT between baseline and 12-months follow-up., Correlations between baseline glycogen levels and change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 12-months follow-up.	—

Measure

Time frame

Change in glycogen concentration of the calf, hamstring, anterior thigh, and lumbar muscles over time – both within the first treatment cycle, and at 3, 6 and 12-months follow-up, Change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 3, 6 and 12-months follow-up., Correlations between baseline glycogen levels and change in FVC between baseline and 12-months follow-up., Correlations between baseline glycogen levels and change in 6MWT between baseline and 12-months follow-up., Correlations between baseline glycogen levels and change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 12-months follow-up.

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Countries

Denmark

Outcome results

None listed