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About 88 results

ALLMedicine™ Alpha-mannosidosis Center

Research & Reviews  30 results

Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis
https://clinicaltrials.gov/ct2/show/NCT02998879

Oct 26th, 2021 - The Primary endpoints of the study include: Safety and tolerability of velmanase alfa as per Adverse events (AEs, including IRR), vital signs, laboratory parameters (hematology, biochemistry and urinanalysis) Detection of anti-velmanase alfa antib...

Alpha-mannosidosis in Tunisian consanguineous families: Potential involvement of varian...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8494324
PloS One; Mkaouar R, Riahi Z et. al.

Oct 7th, 2021 - Alpha-Mannosidosis (AM) is an ultra-rare storage disorder caused by a deficiency of lysosomal alpha-mannosidase encoded by the MAN2B1 gene. Clinical presentation of AM includes mental retardation, recurrent infections, hearing loss, dysmorphic fea...

Alpha-Mannosidosis: A Novel Cause of Bilateral Thalami and Dentate Nuclei Hyperintensity.
https://doi.org/10.1017/cjn.2021.208
The Canadian Journal of Neurological Sciences. Le Journal... Malaquias MJ, Pinto E et. al.

Sep 7th, 2021 - Alpha-Mannosidosis: A Novel Cause of Bilateral Thalami and Dentate Nuclei Hyperintensity.|2021|Malaquias MJ,Pinto E,Oliveira J,Freixo JP,Caseiro C,|

Alpha-mannosidosis in a family: natural history with an uncommon retinal dystrophy.
https://doi.org/10.1097/MCD.0000000000000361
Clinical Dysmorphology; Sandal S, Razdan TB et. al.

Dec 9th, 2020 - Alpha-mannosidosis in a family: natural history with an uncommon retinal dystrophy.|2020|Sandal S,Razdan TB,Verma J,Dubey S,Ghosh A,|

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Clinicaltrials.gov  4 results

Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis
https://clinicaltrials.gov/ct2/show/NCT02998879

Oct 26th, 2021 - The Primary endpoints of the study include: Safety and tolerability of velmanase alfa as per Adverse events (AEs, including IRR), vital signs, laboratory parameters (hematology, biochemistry and urinanalysis) Detection of anti-velmanase alfa antib...

A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis
https://clinicaltrials.gov/ct2/show/NCT01681953

Aug 3rd, 2020 - The overall objective of this trial is to evaluate the efficacy and safety of repeated Lamazym i.v. treatment, compared with placebo, in subjects 5-35 years of age with alpha-Mannosidosis

Safety Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis
https://clinicaltrials.gov/ct2/show/NCT01268358

Aug 3rd, 2020 - This is a single-center, open-label, dose escalation study of patients with alpha-mannosidosis. 10 patients will be enrolled in this study receiving intravenous infusions of Lamazym. In order to avoid development of delayed hypersensitivity all pa...

Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis
https://clinicaltrials.gov/ct2/show/NCT01285700

Sep 26th, 2012 - This is a single-center, open-label, multiple-dose study of the efficacy and long-term safety of Lamazym for the treatment of patients with alpha-mannosidosis.

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News  1 results

EMA Handed Down Fewer Positive Drug Opinions in 2018
https://www.medscape.com/viewarticle/907402

Jan 9th, 2019 - In 2018, the European Medicines Agency (EMA) recommended marketing authorization for 84 new medicines, down from 92 in 2017. The 84 medicines given a thumbs up by EMA's Committee for Medicinal Products for Human Use include 42 agents with a new ac...

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