Bortezomib in the rapid reduction of high sustained antibody titers in disorders treated with therapeutic protein: lessons learned from Pompe disease

Assessing disease severity in Pompe disease: The roles of a urinary glucose tetrasaccharide biomarker and imaging techniques

(2012) Sarah P. Young et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART C-SEMINARS IN MEDICAL GENETICS

Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease

(2012) Yoav H. Messinger et al.   GENETICS IN MEDICINE

The emerging phenotype of long-term survivors with infantile Pompe disease

(2012) Sean N. Prater et al.   GENETICS IN MEDICINE

The impact of antibodies in late-onset Pompe disease: A case series and literature review

(2012) Trusha T. Patel et al.   MOLECULAR GENETICS AND METABOLISM

Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: Need for agents to target antibody-secreting plasma cells

(2012) Suhrad G. Banugaria et al.   MOLECULAR GENETICS AND METABOLISM

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease

(2011) Suhrad G. Banugaria et al.   GENETICS IN MEDICINE

Enzyme replacement therapy induces T-cell responses in late-onset Pompe disease

(2011) Miklos Banati et al.   MUSCLE & NERVE

Therapeutic Potential of Proteasome Inhibition in Duchenne and Becker Muscular Dystrophies

(2010) Elisabetta Gazzerro et al.   AMERICAN JOURNAL OF PATHOLOGY

High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa

(2010) Juna M. de Vries et al.   MOLECULAR GENETICS AND METABOLISM

Bortezomib: a new player in pre- and post-transplant desensitization?

(2010) A. Lemy et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease

(2009) Marc Nicolino et al.   GENETICS IN MEDICINE

Risk factors for inhibitor formation in haemophilia: a prevalent case-control study

(2009) M. V. RAGNI et al.   HAEMOPHILIA

Rituximab and immune tolerance in severe hemophilia A: a consecutive national cohort

(2009) P. W. COLLINS et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

(2009) Priya S. Kishnani et al.   MOLECULAR GENETICS AND METABOLISM

Elimination of Antibodies to Recombinant Enzyme in Pompe's Disease

(2009) Nancy J. Mendelsohn et al.   NEW ENGLAND JOURNAL OF MEDICINE

Early Treatment With Alglucosidase Alfa Prolongs Long-Term Survival of Infants With Pompe Disease

(2009) Priya S Kishnani et al.   PEDIATRIC RESEARCH

Immune response to enzyme replacement therapy in Fabry disease: Impact on clinical outcome?

(2008) C.E.M. Hollak et al.   MOLECULAR GENETICS AND METABOLISM

Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment

(2008) Jinhai Wang et al.   NATURE BIOTECHNOLOGY

The proteasome inhibitor bortezomib depletes plasma cells and protects mice with lupus-like disease from nephritis

(2008) Kirsten Neubert et al.   NATURE MEDICINE