human GALNS protein

BMN-100 and BMN 110 are recombinant GALNS; RefSeq NM_000512

Also Known As:

GALNS protein, human; BMN 110; BMN-100; GALNAC6S protein, human; Morquio syndrome protein, human; N-acetylgalactosamine-6-sulfatase, human; Vimizim; elosulfase alfa; galactosamine (N-acetyl)-6-sulfate sulfatase, human; galactose-6-sulfate sulfatase, human; mucopolysaccharidosis type IVA protein, human

Networked: 31 relevant articles (7 outcomes, 15 trials/studies)

Relationship Network

Bio-Agent Context: Research Results

Enzymes and Coenzymes: 1
- Enzymes: 152586
  - Hydrolases: 2166
    - Esterases: 1574
      - Sulfatases: 574
        Chondroitinases and Chondroitin Lyases: 108
        Chondroitinsulfatases
        human GALNS protein: 31

Experts

1.	Harmatz, Paul: 6 articles (01/2017 - 11/2014)
2.	Giugliani, Roberto: 4 articles (01/2018 - 11/2014)
3.	Hendriksz, Christian J: 4 articles (01/2018 - 11/2014)
4.	Jones, Simon A: 4 articles (01/2018 - 11/2014)
5.	Guelbert, Norberto: 3 articles (03/2021 - 08/2015)
6.	Hughes, Derralynn: 3 articles (01/2021 - 11/2014)
7.	Tomatsu, Shunji: 3 articles (10/2019 - 11/2005)
8.	Decker, Celeste: 3 articles (01/2018 - 01/2017)
9.	Jesaitis, Lynne: 3 articles (01/2018 - 05/2015)
10.	Parini, Rossella: 3 articles (01/2018 - 02/2015)

Related Diseases

1.	Mucopolysaccharidosis IV (Morquio Syndrome) 10/01/2014 - "In the key placebo-controlled, 24-week, phase 3 trial in patients with MPS IVA aged ≥5 years, elosulfase alfa 2 mg/kg/week significantly improved endurance [least squares mean placebo-adjusted change from baseline in 6-min walk test distance 22.5 m (95 % CI 4.0-40.9)]. " 06/01/2018 - "Our results showed overall improvement in endurance and functionality after 8 months of elosulfase alfa treatment in a heterogeneous subset of MPS IVA patients with severe clinical manifestations managed in a real-world setting." 11/01/2015 - "The study was designed to evaluate safety and efficacy of elosulfase alfa in patients with MPS IVA aged 5 years and older. " 08/20/2022 - "Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis." 09/01/2022 - "Findings from the Morquio A Registry Study (MARS) after 6 years: Long-term outcomes of MPS IVA patients treated with elosulfase alfa."
2.	Mucopolysaccharidoses 01/01/2017 - "Name of registry: Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome). " 02/01/2015 - "To report and discuss the multi-domain impact of elosulfase alfa, with focus on tertiary and composite endpoints, in the 24-week, randomized, double-blind, placebo-controlled phase 3 trial in patients with Morquio A syndrome (mucopolysaccharidosis IVA). " 01/01/2016 - "Role of elosulfase alfa in mucopolysaccharidosis IVA." 12/01/2015 - "Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y." 01/01/2018 - "Long-term safety and efficacy of elosulfase alfa enzyme replacement therapy (ERT) were assessed in 173 patients with Morquio A syndrome (mucopolysaccharidosis IVA) in a 96-week, open-label, multi-center, phase 3 extension study (MOR-005) of the pivotal 24-week, placebo-controlled study (MOR-004). "
3.	Rare Diseases (Rare Disease) 01/01/2017 - "The objective of the present study was to review minimal clinically important differences (MCIDs) for the 6MWT reported for disease states that widely use the 6MWT to evaluate clinical benefit and to discuss the results in view of the challenges in estimating MCID for ultra-rare diseases, using the case of elosulfase alfa in Morquio A patients. " 08/01/2015 - "Data from the ongoing observational, multinational Morquio A Registry Study will provide valuable information on the long-term efficacy and safety of elosulfase alfa in patients, as well as on the natural history of this very rare disease."
4.	Pain (Aches) 10/01/2015 - "In conclusion, preliminary data of this small study in a Morquio A population with relatively good endurance confirmed the acceptable safety profile of elosulfase alfa and showed a trend of increased exercise capacity and muscle strength and decreased pain." 01/01/2021 - "This managed access agreement enabled the collection of comprehensive real-world data for patients with MPS IVA, with results demonstrating that patients starting elosulfase alfa treatment showed gains similar to those seen in the pivotal trial for outcomes including endurance, respiratory and cardiac function, pain, quality of life measures and urinary keratan sulfate levels. "
5.	Lysosomal Storage Diseases (Lysosomal Storage Disease) 10/11/2019 - "Enzyme-Loaded Gel Core Nanostructured Lipid Carriers to Improve Treatment of Lysosomal Storage Diseases: Formulation and In Vitro Cellular Studies of Elosulfase Alfa-Loaded Systems."

Related Drugs and Biologics

1.	human GALNS protein
2.	Enzymes
3.	Keratan Sulfate
4.	Biomarkers (Surrogate Marker)
5.	Lipids
6.	Droxidopa
7.	Sulfates (Sulfates, Inorganic)
8.	Sulfatases
9.	Phosphates (Orthophosphate)
10.	Keratins (Keratin)

Related Therapies and Procedures

1.	Enzyme Replacement Therapy
2.	Therapeutics