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About 115 results

ALLMedicine™ Alpha-mannosidosis Center

Research & Reviews  36 results

European Alpha-Mannosidosis Participant
https://clinicaltrials.gov/ct2/show/NCT03651245

Jun 13th, 2022 - Alpha-Mannosidosis is a lysosomal storage disorder of the Glycoprotein family of diseases and is closely related to Mucopolysaccharidoses.It is a rare inherited disorder that causes problems in many organs and tissues of the body. Affected individ...

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,|

Biomarker for Mannosidosis Disease (BioMannosidosis)
https://clinicaltrials.gov/ct2/show/NCT03264040

May 13th, 2021 - Alpha-Mannosidosis is a rare lysosomal storage disorder of the Glycoprotein family of diseases and is closely related to Mucopolysaccharidoses. Alpha-Mannosidosis was first described by Dr Oekerman, from Lund in Sweden in 1967. There is another va...

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Clinicaltrials.gov  9 results

European Alpha-Mannosidosis Participant
https://clinicaltrials.gov/ct2/show/NCT03651245

Jun 13th, 2022 - Alpha-Mannosidosis is a lysosomal storage disorder of the Glycoprotein family of diseases and is closely related to Mucopolysaccharidoses.It is a rare inherited disorder that causes problems in many organs and tissues of the body. Affected individ...

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...

Biomarker for Mannosidosis Disease (BioMannosidosis)
https://clinicaltrials.gov/ct2/show/NCT03264040

May 13th, 2021 - Alpha-Mannosidosis is a rare lysosomal storage disorder of the Glycoprotein family of diseases and is closely related to Mucopolysaccharidoses. Alpha-Mannosidosis was first described by Dr Oekerman, from Lund in Sweden in 1967. There is another va...

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...

The Natural History of Alpha-Mannosidosis
https://clinicaltrials.gov/ct2/show/NCT00498420

Aug 3rd, 2020 - Definition: Human alpha-mannosidosis is a rare genetic disorder, caused by the lack of lysosomal alpha-mannosidase, resulting in mental retardation, skeletal changes, hearing loss and recurrent infections. The lack of alpha-mannosidase causes a di...

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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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