Cargando…

Multicenter, non-interventional, double cohort study to assess the safety of alglucosidase alfa and laronidase in real-world home infusion setting

Detalles Bibliográficos
Autores principales:	Toscano, Antonio, Scarpa, Maurizio, Parini, Rossella, Donati, Alice, Crescimanno, Grazia, Fiumara, Agata, Fischetto, Rita, Gasperini, Serena, Siciliano, Gabriele, Ravaglia, Sabrina, Sechi, Annalisa, Piperno, Alberto, Cianci, Vittorio, Maggi, Lorenzo, Musumeci, Olimpia, Taurisano, Roberta, Verrecchia, Elena
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Academic Press 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8811529/ http://dx.doi.org/10.1016/j.ymgme.2021.11.319

Ejemplares similares

Safety outcomes and patients’ preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond
por: Toscano, Antonio, et al.
Publicado: (2023)

STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa
por: Gutschmidt, Kristina, et al.
Publicado: (2021)

Alglucosidase alfa: Long term use in the treatment of patients with Pompe disease
por: Beck, Michael
Publicado: (2009)

Alglucosidase alfa: 5 years of experience in late-onset Pompe disease
por: Schoser, Benedikt
Publicado: (2013)

Long-Term Experience with Anaphylaxis and Desensitization to Alglucosidase Alfa in Pompe Disease
por: Ertoy Karagol, Hacer Ilbilge, et al.
Publicado: (2023)

Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel
por: Gragnaniello, Vincenza, et al.
Publicado: (2022)

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease
por: Kanters, Tim A., et al.
Publicado: (2017)

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease
por: Kanters, Tim A, et al.
Publicado: (2014)

Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV
por: Yi, Haiqing, et al.
Publicado: (2016)

Novel approach to idursulfase and laronidase desensitization in type 2 and type 1 S mucopolysaccharidosis (MPS)
por: Spataro, Federico, et al.
Publicado: (2022)

A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa in Chinese Patients With Infantile-Onset Pompe Disease
por: Zhu, Diqi, et al.
Publicado: (2022)

Effects of higher and more frequent dosing of alglucosidase alfa and immunomodulation on long‐term clinical outcome of classic infantile Pompe patients
por: Poelman, Esther, et al.
Publicado: (2020)

Double dose alglucosidase-alpha doubles benefit?
por: Finsterer, Josef, et al.
Publicado: (2018)

Earlier and higher dosing of alglucosidase alfa improve outcomes in patients with infantile-onset Pompe disease: Evidence from real-world experiences
por: Chien, Yin-Hsiu, et al.
Publicado: (2020)

Immune tolerance induction for laronidase treatment in mucopolysaccharidosis I
por: Giugliani, Roberto, et al.
Publicado: (2017)

The impact of laronidase treatment in otolaryngological manifestations of patients with mucopolysaccharidosis()
por: Dualibi, Ana Paula Fiuza Funicello, et al.
Publicado: (2015)

The influence of a polymorphism in the gene encoding angiotensin converting enzyme (ACE) on treatment outcomes in late-onset Pompe patients receiving alglucosidase alfa
por: Baek, Rena C., et al.
Publicado: (2016)

Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions
por: Ditters, Imke A. M., et al.
Publicado: (2023)

Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes
por: Lund, Troy C., et al.
Publicado: (2019)

Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation
por: Polgreen, Lynda E., et al.
Publicado: (2019)

Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study
por: van Gelder, C. M., et al.
Publicado: (2016)

I-4 Long-term follow-up effects on enzyme replacement treatment of adult form of acid maltase deficiency myopathy
por: Angelini, C., et al.
Publicado: (2011)

Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis
por: Dornelles, Alícia Dorneles, et al.
Publicado: (2017)

Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy
por: Parini, Rossella, et al.
Publicado: (2018)

Data from subjects receiving intrathecal laronidase for cervical spinal stenosis due to mucopolysaccharidosis type I
por: Dickson, P.I., et al.
Publicado: (2015)

CASE SERIES OF PATIENTS UNDER BIWEEKLY TREATMENT WITH LARONIDASE: A REPORT OF A SINGLE CENTER EXPERIENCE
por: Kyosen, Sandra Obikawa, et al.
Publicado: (2019)

Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
por: Horovitz, Dafne Dain Gandelman, et al.
Publicado: (2016)

Transcranial pulsed ultrasound facilitates brain uptake of laronidase in enzyme replacement therapy for Mucopolysaccharidosis type I disease
por: Hsu, Yu-Hone, et al.
Publicado: (2017)

S3.1 Are there ERT defined guidelines for Pompe disease?
por: Toscano, Antonio, et al.
Publicado: (2011)

S2.3 Therapeutic approaches in the late onset form of GSD II
por: Musumeci, Olimpia, et al.
Publicado: (2011)

Early is better? A new algorithm for early diagnosis in Late Onset Pompe Disease (LOPD)
por: TOSCANO, ANTONIO, et al.
Publicado: (2013)

Mis-splicing of the GALNS gene resulting from deep intronic mutations as a cause of Morquio a disease
por: Caciotti, Anna, et al.
Publicado: (2018)

Real-world data assessment of safety of home-based and hospital/outpatient-based laronidase enzyme replacement therapy for mucopolysaccharidosis I
por: Wilson, Amanda, et al.
Publicado: (2021)

Statins Neuromuscular Adverse Effects
por: Attardo, Silvia, et al.
Publicado: (2022)

Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y
por: Jones, Simon A., et al.
Publicado: (2015)

O-3 Clinical features and outcome measures during 1 year enzyme replacement therapy in late onset GSD II patients
por: Angelini, C., et al.
Publicado: (2011)

Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships
por: Al-Sannaa, Nouriya A., et al.
Publicado: (2015)

Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α
por: Kishnani, Priya, et al.
Publicado: (2017)

Analysis of Renal and Cardiac Outcomes in Male Participants in the Fabry Outcome Survey Starting Agalsidase Alfa Enzyme Replacement Therapy Before and After 18 Years of Age
por: Parini, Rossella, et al.
Publicado: (2020)

Molecular Genetics Overview of Primary Mitochondrial Myopathies
por: Arena, Ignazio Giuseppe, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_bafko2eli5dk2poe9oa4hj3kpi