Bone marrow transplantation in the treatment of alpha-mannosidosis. Arch. Dis. Childh 62:1044-1049.Will A, Cooper A, Hatton C, Sardharwalla IB, Evans DI, Stevens RF: Bone marrow transplantation in the treatment of alpha- mannosidosis. Arch Dis Child 1987, 62:1044-1049....
Specific treatment Inα-mannosidosis all cells are devoid ofα-mannosidase activity. Early observations showed that cells producingα-mannosidase were able to transfer the enzyme to mannosidosis cells [63,66]. The rationale for bone marrow transplantation (BMT) in mannosidosis is that enzyme-pr...
“As someone who knows personally how devastating alpha-mannosidosis can be, this is an important milestone toward a potential first treatment option that addresses the root cause of the disease and is bringing patients, parents, families and caregivers hope for a bett...
Oligosaccharide levels appeared correlated with functional testing and may serve as biomarkers of disease severity, progression and response to treatment. Trial registration ClinicalTrials.gov Identifier = NCT00498420 and EuropeanCommission FP VI contract LHSM-CT-2006-018692....
In this report, we describe the process of alpha-mannosidosis diagnosis and treatment from the caregivers’ and physicians’ perspectives. For the caregivers’ perspective, the mothers of two patients with alpha-mannosidosis (‘Adele’ aged 35 years and ‘Amedeo’ aged 40 years) were ...
Effective treatment of α-mannosidosis by allogeneic hematopoietic stem cell transplantation To study the efficacy of hematopoietic stem cell transplantation (HCT) for ameliorating the clinical manifestations of α-mannosidosis. Four patients with ... SS Grewal,EG Shapiro,W Krivit,... - 《J Pedia...
In cats with α-mannosidosis induced by swainsonine, some pyramidal neurons showed GM2-like immunoreactivity after 4 weeks of treatment, whereas ectopic dendrites only became evident after 7 weeks of treatment. Thus, GM2 ganglioside accumulated in pyramidal neurons before ectopic dendrites emerged ...
While bone marrow transplantation has been the only therapeutic option to date, nowadays new treatment options are being explored, which may affect pulmonary and exercise capacity. Aim and Methods To assess cardiopulmonary involvement in patients with α mannosidosis by pulmonary function tests, ...
Our data demonstrate early neuropathological changes, which may be taken into consideration when planning initiation of treatment.L.DepartmentBorgwardtDepartmentE.R.DepartmentDanielsenDepartmentC.DepartmentThomsenDepartmentJ.E.DepartmentMånssonDepartment
Recently, enzyme replacement therapy was approved in the European Union for the treatment of alpha-mannosidosis, but evaluation regarding long-term efficacy and safety is hard to assess due to missing quantitative natural history data, in particular survival. Methods: We performed a quantitative ...