Health Related Quality of Life, Disability, and Pain in Alpha Mannosidosis: Long-Term Data of Enzyme Replacement Therapy With Velmanase Alfa (Human Recombinant Alpha Mannosidase)

Borgwardt, Line; Guffon, Nathalie; Amraoui, Yasmina; Jones, Simon A.; Meirleir, Linda De; Lund, Allan M.; Gil-Campos, Mercedes; Hout, Johanna M. P. Van den; Tylki-Szymanska, Anna; Geraci, Silvia; Ardigò, Diego; Cattaneo, Federica; Harmatz, Paul; Phillips, Dawn

doi:10.1177/2326409818796854

Line Borgwardt

Centre for Inherited Metabolic Diseases, Department of Paediatrics and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark

Center for Genomic Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark

Nathalie Guffon

Centre de Référence des Maladies Héréditaires du Métabolisme, Hôpital Femme Mère Enfant, Lyon, France

Yasmina Amraoui

University Medical Centre Mainz, Centre for Pediatric and Adolescent Medicine, Villa Metabolica, Mainz, Germany

Simon A. Jones

Manchester Centre for Genomic Medicine, St. Mary’s Hospital, Central Manchester University Hospitals NHS Foundation Trust, Manchester Academic Health Sciences Centre, Manchester, United Kingdom

Linda De Meirleir

Paediatric Neurology and Metabolism, Universitair Ziekenhuis, Brussels, Belgium

Allan M. Lund

Centre for Inherited Metabolic Diseases, Departments of Paediatrics and Clinical Genetics, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark

Mercedes Gil-Campos

Unidad de Metabolismo e Investigación Pediátrica, Hospital Reina Sofía, Córdoba, Spain

Johanna M. P. Van den Hout

Center for Lysosomal and Metabolic Diseases, Erasmus Medical Center—Sophia Children’s Hospital, Rotterdam, the Netherlands

Anna Tylki-Szymanska

Department of Paediatric, Nutrition and Metabolic Diseases, The Children’s Memorial Health Institute, Warsaw, Poland

Silvia Geraci

Chiesi Farmaceutici S.p.A, Parma, Italy

Declaration of Conflicting Interests The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: S.G., D.A., and F.C. are employees of Chiesi Farmaceutici S.p.A., sponsor of the rhLAMAN-10 study and this resulting manuscript. Chiesi’s subsidiary, Zymenex, is developing velmanase alfa for treatment of alpha-mannosidosis. D.P. is a salaried employee of Evidera, a research and consulting company for the biopharmaceutical industry; she does not accept remuneration of any sort from Evidera clients. Chiesi Farmaceutici S.p.A contracted with Evidera for writing assistance on this manuscript. She reports receiving consulting fees from Chiesi prior to employment at Evidera.

Diego Ardigò

Chiesi Farmaceutici S.p.A, Parma, Italy

Declaration of Conflicting Interests The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: S.G., D.A., and F.C. are employees of Chiesi Farmaceutici S.p.A., sponsor of the rhLAMAN-10 study and this resulting manuscript. Chiesi’s subsidiary, Zymenex, is developing velmanase alfa for treatment of alpha-mannosidosis. D.P. is a salaried employee of Evidera, a research and consulting company for the biopharmaceutical industry; she does not accept remuneration of any sort from Evidera clients. Chiesi Farmaceutici S.p.A contracted with Evidera for writing assistance on this manuscript. She reports receiving consulting fees from Chiesi prior to employment at Evidera.

Federica Cattaneo

Chiesi Farmaceutici S.p.A, Parma, Italy

Declaration of Conflicting Interests The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: S.G., D.A., and F.C. are employees of Chiesi Farmaceutici S.p.A., sponsor of the rhLAMAN-10 study and this resulting manuscript. Chiesi’s subsidiary, Zymenex, is developing velmanase alfa for treatment of alpha-mannosidosis. D.P. is a salaried employee of Evidera, a research and consulting company for the biopharmaceutical industry; she does not accept remuneration of any sort from Evidera clients. Chiesi Farmaceutici S.p.A contracted with Evidera for writing assistance on this manuscript. She reports receiving consulting fees from Chiesi prior to employment at Evidera.

Paul Harmatz

UCSF Benioff Children’s Hospital Oakland, Oakland, CA, USA

Dawn Phillips Corresponding Author: Dawn Phillips, One Kendall Square, Building 300, Suite 201, Cambridge, MA 02139, US. Email: DawnPhillipspt@gmail.com

Evidera, Bethesda, MD, USA

Declaration of Conflicting Interests The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: S.G., D.A., and F.C. are employees of Chiesi Farmaceutici S.p.A., sponsor of the rhLAMAN-10 study and this resulting manuscript. Chiesi’s subsidiary, Zymenex, is developing velmanase alfa for treatment of alpha-mannosidosis. D.P. is a salaried employee of Evidera, a research and consulting company for the biopharmaceutical industry; she does not accept remuneration of any sort from Evidera clients. Chiesi Farmaceutici S.p.A contracted with Evidera for writing assistance on this manuscript. She reports receiving consulting fees from Chiesi prior to employment at Evidera.

Corresponding Author: Dawn Phillips, One Kendall Square, Building 300, Suite 201, Cambridge, MA 02139, US. Email: DawnPhillipspt@gmail.com

Declaration of Conflicting Interests

The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: S.G., D.A., and F.C. are employees of Chiesi Farmaceutici S.p.A., sponsor of the rhLAMAN-10 study and this resulting manuscript. Chiesi’s subsidiary, Zymenex, is developing velmanase alfa for treatment of alpha-mannosidosis. D.P. is a salaried employee of Evidera, a research and consulting company for the biopharmaceutical industry; she does not accept remuneration of any sort from Evidera clients. Chiesi Farmaceutici S.p.A contracted with Evidera for writing assistance on this manuscript. She reports receiving consulting fees from Chiesi prior to employment at Evidera.