Enzyme Replacement Therapy (ert) - Neonatal Disorders

Relevant Publications

Plasma and platelet lipidome changes in Fabry disease.

Issue Release: 2024

Laronidase-loaded liposomes reach the brain and other hard-to-treat organs after noninvasive nasal administration.

Issue Release: 2024

Clinical and preclinical insights into high-dose ambroxol therapy for Gaucher disease type 2 and 3: A comprehensive systematic review.

Issue Release: 2024

Start, switch and stop (triple-S) criteria for enzyme replacement therapy of late-onset Pompe disease: European Pompe Consortium recommendation update 2024.

Issue Release: 2024

Fabry disease: development and progression of left ventricular hypertrophy despite long-term enzyme replacement therapy.

Issue Release: 2024

Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy.

Issue Release: 2024

Advances in Immune Tolerance Induction in Enzyme Replacement Therapy.

Issue Release: 2024

Treatment with Elapegademase Restores Immunity in Infants with Adenosine Deaminase Deficient Severe Combined Immunodeficiency.

Issue Release: 2024

Immobilization of human tyrosine hydroxylase onto magnetic nanoparticles - A novel formulation of a therapeutic enzyme.

Issue Release: 2024

Effects of Enzyme Replacement Therapy on Cardiac MRI Findings in Fabry Disease: A Systematic Review and Meta-Analysis.

Issue Release: 2024

Rapidly progressive cognitive impairment resulting in heavy psychosocial burden in a patient with Fabry disease undergoing hemodialysis: a case report.

Issue Release: 2024

Impact of COVID-19 pandemic on healthcare delivery for lysosomal storage disorders at a tertiary care public hospital in Mumbai.

Issue Release: 2024

Complement activation and cellular inflammation in Fabry disease patients despite enzyme replacement therapy.

Issue Release: 2024

Efficacy and safety of enzyme replacement therapy with alglucosidase alfa for the treatment of patients with infantile-onset Pompe disease: a systematic review and metanalysis.

Issue Release: 2024

104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).

Issue Release: 2024

Long-term clinical evaluation of patients with alpha-mannosidosis - A multicenter study.

Issue Release: 2024

Updated Evaluation of Agalsidase Alfa Enzyme Replacement Therapy for Patients with Fabry Disease: Insights from Real-World Data.

Issue Release: 2024

Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease.

Issue Release: 2024

Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data.

Issue Release: 2024

Diet and Physical Activity in Fabry Disease: A Narrative Review.

Issue Release: 2024

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