Methylmalonic acidemia/propionic acidemia – the biochemical presentation and comparing the outcome between liver transplantation versus non-liver transplantation groups

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

(2017) Ding An et al.   Cell Reports

Improvement in the prognosis and development of patients with methylmalonic acidemia after living donor liver transplant

(2016) Rieko Sakamoto et al.   PEDIATRIC TRANSPLANTATION

Juvenile gout in methylmalonic acidemia

(2016) Sirirat Charuvanij et al.   PEDIATRICS INTERNATIONAL

Minimization or withdrawal of immunosuppressants in pediatric liver transplant recipients

(2015) Niang-Cheng Lin et al.   JOURNAL OF PEDIATRIC SURGERY

Liver transplantation in severe methylmalonic acidemia: The sooner, the better

(2015) Marco Spada et al.   JOURNAL OF PEDIATRICS

Treatment of Methylmalonic Acidemia by Liver or Combined Liver-Kidney Transplantation

(2015) Anna-Kaisa Niemi et al.   JOURNAL OF PEDIATRICS

Liver or Combined Liver-Kidney Transplantation for Patients with Isolated Methylmalonic Acidemia: Who and When?

(2015) Jennifer L. Sloan et al.   JOURNAL OF PEDIATRICS

Cost-effectiveness of liver transplantation in methylmalonic and propionic acidemias

(2015) Meng Li et al.   LIVER TRANSPLANTATION

Reversal of cardiomyopathy in propionic acidemia after liver transplantation: a 10-year follow-up

(2015) Chiara Arrizza et al.   TRANSPLANT INTERNATIONAL

Clinical features and MUT gene mutation spectrum in Chinese patients with isolated methylmalonic acidemia: identification of ten novel allelic variants

(2015) Lian-Shu Han et al.   World Journal of Pediatrics

A detailed analysis of methylmalonic acid kinetics during hemodialysis and after combined liver/kidney transplantation in a patient with mut 0 methylmalonic acidemia

(2014) Hilary J. Vernon et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Liver transplantation for pediatric metabolic disease

(2014) George Mazariegos et al.   MOLECULAR GENETICS AND METABOLISM

Proposed guidelines for the diagnosis and management of methylmalonic and propionic acidemia

(2014) Matthias R Baumgartner et al.   Orphanet Journal of Rare Diseases

Monogenic diseases that can be cured by liver transplantation

(2013) Stefano Fagiuoli et al.   JOURNAL OF HEPATOLOGY

Pediatric liver transplantation for urea cycle disorders and organic acidemias: United Network for Organ Sharing data for 2002-2012

(2013) Emily R. Perito et al.   LIVER TRANSPLANTATION

Long-term neurological outcome of a cohort of 80 patients with classical organic acidurias

(2013) Mathilde Nizon et al.   Orphanet Journal of Rare Diseases

Living donor liver transplantation for pediatric patients with metabolic disorders: The Japanese multicenter registry

(2013) Mureo Kasahara et al.   PEDIATRIC TRANSPLANTATION

Cardiomyopathies in Propionic Aciduria are Reversible After Liver Transplantation

(2009) Stéphane Romano et al.   JOURNAL OF PEDIATRICS

Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy

(2009) Randy J Chandler et al.   MOLECULAR THERAPY

Long-term outcome following pediatric liver transplantation for metabolic disorders

(2009) Terrell Stevenson et al.   PEDIATRIC TRANSPLANTATION