Evaluation of 2 patients with alpha-mannosidosis and history of conductive hearing impairment participating in a placebo-controlled, phase 3 program receiving velmanase alfa (human recombinant alpha-mannosidase)

被引：0
|
作者：
Lund, Allan [1 ]
Borgwardt, Line Gutte [2 ]
Regal, Luc [3 ]
Cattaneo, Frederica [4 ]
机构：
[1] Copenhagen Univ Hosp, Rigshosp, Ctr Inherited Metab Dis, Copenhagen, Denmark
[2] Copenhagen Univ Hosp, Rigshosp, Ctr Inherited Metab Dis, Ctr Genom Med, Copenhagen, Denmark
[3] Univ Ziekenhuis, Brussels, Belgium
[4] Chiesi Farmaceut SpA, Parma, Italy
来源：
MOLECULAR GENETICS AND METABOLISM | 2021年 / 132卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2020.12.152
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
146
引用
收藏
页码：S67 / S67
页数：1
相关论文
共 11 条
  • [1] A new randomized placebo-controlled study to establish the safety and efficacy of velmanase alfa (human recombinant alpha-mannosidase) enzyme replacement therapy for the treatment of alpha-mannosidosis
    Harmatz, Paul
    Jayakar, Parul
    Pena, Loren D. M.
    Cattaneo, Federica
    Ceravolo, Ferdinando
    Paleari, Ylenia
    Sutton, Vernon R.
    Burton, Barbara K.
    Longo, Nicola
    [J]. MOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S70 - S70
  • [2] Long-term enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase) improves mobility in alpha-mannosidosis patients
    Lund, Allan M.
    Borgwardt, Line
    Amraoui, Yasmina
    Andersen, Oluf
    De Meirleir, Linda
    Dolhem, Philippe
    Gil Campos, Mercedes
    Guffon, Nathalie
    Heron, Benedicte
    Jameson, Elisabeth
    Jones, Simon A.
    Laroche, Cecile
    Lindberg, Christoffer
    Marquardt, Thorsten
    Mengel, Karl-Eugene
    Muschol, Nicole
    Tylki-Szymanska, Anna
    Van den Hout, Johanna M. P.
    Van der Ploeg, Ans
    Welling, Lindsey
    Wijburg, Frits
    Fogh, Jens
    Geraci, Silvia
    Ardigo, Diego
    Cattaneo, Federica
    [J]. MOLECULAR GENETICS AND METABOLISM, 2017, 120 (1-2) : S88 - S89
  • [3] Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis
    Harmatz, Paul
    Cattaneo, Federica
    Ardigo, Diego
    Geraci, Silvia
    Hennermann, Julia B.
    Guffon, Nathalie
    Lund, Allan
    Hendriksz, Christian J.
    Borgwardt, Line
    [J]. MOLECULAR GENETICS AND METABOLISM, 2018, 124 (02) : 152 - 160
  • [4] Long-term enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase) slows disease progression in adult patients suffering from alpha-mannosidosis
    Borgwardt, Line
    Lund, Allan M.
    Amraoui, Yasmina
    Andersen, Oluf
    De Meirleird, Linda
    Dolhem, Philippe
    Campos, Mercedes Gil
    Guffon, Nathalie
    Heron, Benedicte
    Laroche, Cecile
    Lindberg, Christoffer
    Marquardt, Thorsten
    Mengel, Karl-Eugene
    Muschol, Nicole
    Tylki-Szymanska, Anna
    Van Den Hout, Johanna M. P.
    Van Der Ploeg, Ans
    Welling, Lindsey
    Wijburg, Frits
    Cole, Duncan
    Fogh, Jens
    Geraci, Silvia
    Ardigo, Diego
    Cattaneo, Federica
    [J]. MOLECULAR GENETICS AND METABOLISM, 2017, 120 (1-2) : S30 - S30
  • [5] Improvement in pulmonary function and serum immunoglobulin G in long-term enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase) in alpha-mannosidosis patients
    Borgwardt, Line
    Lund, Allan M.
    De Meirleir, Linda
    Amraoui, Yasmina
    Andersen, Oluf
    Dolhem, Philippe
    Campos, Mercedes Gil
    Guffon, Nathalie
    Heron, Benedicte
    Jameson, Elisabeth
    Jones, Simon A.
    Laroche, Cecile
    Lindberg, Christoffer
    Marquardt, Thorsten
    Mengel, Karl-Eugene
    Muschol, Nicole
    Van den Hout, Johanna M. P.
    Welling, Lindsey
    Wijburg, Frits
    Cole, Duncan
    Van der Ploeg, Ans
    Tylki-Szymanska, Anna
    Fogh, Jens
    Geraci, Silvia
    Ardigo, Diego
    Cattaneo, Federica
    [J]. MOLECULAR GENETICS AND METABOLISM, 2017, 120 (1-2) : S29 - S29
  • [6] The first study investigating safety and efficacy of velmanase alfa (human recombinant alpha mannosidase) in alpha-mannosidosis patients below six years of age
    Guffon, Nathalie
    Muschol, Nicole M.
    Konstantopoulou, Vassiliki
    Cattaneo, Federica
    Gelmetti, Elisabetta
    Tummolo, Albina
    Bruno, Irene
    Borgwardt, Line
    Hennermann, Julia B.
    Lund, Allan M.
    [J]. MOLECULAR GENETICS AND METABOLISM, 2019, 126 (02) : S65 - S65
  • [7] Effect of velmanase alfa (human recombinant alpha-mannosidase) enzyme-replacement therapy on quality of life and disease burden of patients with alpha-mannosidosis: Results from caregiver feedback
    Lund, Allan
    Guffon, Nathalie
    Gil-Campos, Mercedes
    Cattaneo, Frederica
    Heron, Benedicte
    Tylki-Szymanska, Anna
    Borgwardt, Line
    Regal, Luc
    Cole, Duncan
    Hennermann, Julia
    [J]. MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S67 - S67
  • [8] Improvement in fine and gross motor proficiency after long-term enzyme replacement therapy with velmanase alfa (human recombinant alpha mannosidase) in alpha-mannosidosis patients
    Borgwardt, Line
    Lund, Allan M.
    Amraoui, Yasmine
    Andersen, Oluf
    De Meirleir, Linda
    Dolhem, Philippe
    Campos, Mercedes Gil
    Guffon, Nathalie
    Heron, Benedicte
    Jameson, Elisabeth
    Jones, Simon A.
    Laroche, Cecile
    Lindberg, Christoffer
    Marquardt, Thorsten
    Mengel, Karl-Eugene
    Muschol, Nicole
    Tylki-Szymanska, Anna
    Van den Hout, Johanna M. P.
    Van der Ploeg, Ans
    Welling, Lindsey
    Wijburg, Frits
    Fogh, Jens
    Geraci, Silvia
    Ardigo, Diego
    Cattaneo, Federica
    Phillips, Dawn
    [J]. MOLECULAR GENETICS AND METABOLISM, 2017, 120 (1-2) : S29 - S30
  • [9] Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial
    Borgwardt, Line
    Guffon, Nathalie
    Amraoui, Yasmina
    Dali, Christine I.
    De Meirleir, Linda
    Gil-Campos, Mercedes
    Heron, Benedicte
    Geraci, Silvia
    Ardigo, Diego
    Cattaneo, Federica
    Fogh, Jens
    Van den Hout, J. M. Hannerieke
    Beck, Michael
    Jones, Simon A.
    Tylki-Szymanska, Anna
    Haugsted, Ulla
    Lund, Allan M.
    [J]. JOURNAL OF INHERITED METABOLIC DISEASE, 2018, 41 (06) : 1215 - 1223
  • [10] AIPAC-003: A randomized, double-blind, placebo-controlled phase 3 trial testing eftilagimod alpha (soluble LAG-3) in patients with HER2-neg/low metastatic breast cancer receiving paclitaxel, following an open-label dose optimization
    Forget, Frederic
    Murillo, Serafin Morales
    Ibrahim, Nuhad K.
    Uribe, Bernard Doger de Speille
    Marathe, Omkar
    Canon, Jean-Luc Re
    Chalasani, Pavani
    Papadimitriou, Konstantinos
    Oliveira, Mafalda
    Segui, Elia
    Triebel, Frederic
    [J]. JOURNAL OF CLINICAL ONCOLOGY, 2024, 42 (16)
12