The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamzede i.v. treatment of subjects with alpha-Mannosidosis.
This trial provide aftercare treatment with Lamzede to French patients.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
13
ERT, i.v. infusions weekly
Hôpital Femme Mére Enfant - CHU de Lyon
Bron, France
Change from baseline in Adverse events
AE documented at every visit
Time frame: 3 year
Change from baseline in Lamazym antibodies
AB measured every 12th week
Time frame: 3 year
progress from baseline in number of steps climbed in 3 minutes
Time frame: 1 year, 2 year and 3 year
Progress from baseline in equivalent age
Time frame: 1 year, 2 year and 3 year
Progress from baseline in Forced Vital Capacity
Time frame: 1 year, 2 year and 3 year
Progress from baseline in distance walked in 6 minutes
Time frame: 1 year, 2 year and 3 year
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