Study on the Effectiveness and Safety of Nizubaglustat in Patients with Niemann-Pick Type C, GM1, or GM2 Gangliosidosis
- Substances:NIZUBAGLUSTAT
Portugal- Participants:0–17 years · 18–64 years
- Substances:NIZUBAGLUSTAT
- Sponsor:Azafaros B.V.
Rare diseases
Investigational molecules
Locations
Back to Portugal638 clinical trials in Lisbon, Portugal across 3 research sites. Every trial is listed in the EU Clinical Trials Register and updated daily.
Our site in PortugalEnsaios-Clinicos.pt(opens in a new tab)
PortugalStudy on Acoramidis for Preventing Transthyretin Amyloidosis in Young People with a Genetic Risk
PortugalStudy on the Safety and Effectiveness of Inclisiran for Children Aged 6 to Under 12 with Familial Hypercholesterolemia
PortugalStudy on Abatacept for Predicting Treatment Response in Patients with Early Rheumatoid Arthritis
PortugalStudy on the Effectiveness and Safety of Ianalumab for Patients with Diffuse Cutaneous Systemic Sclerosis
PortugalStudy on the Effectiveness and Safety of Vedolizumab and Upadacitinib in Adults with Moderate to Severe Crohn's Disease
PortugalStudy on the Effectiveness of Gilteritinib and Drug Combination for Patients with Relapsed or Refractory Acute Myeloid Leukemia
PortugalStudy on Frexalimab, SAR442970, and Rilzabrutinib for Patients Aged 16-75 with Focal Segmental Glomerulosclerosis or Minimal Change Disease
PortugalStudy on Ifinatamab Deruxtecan for Patients with Recurrent or Metastatic Solid Tumors
PortugalStudy of Sarilumab for Children and Adolescents with Systemic Juvenile Idiopathic Arthritis (sJIA)
PortugalStudy of Mirikizumab for Children with Moderate to Severe Crohn's Disease
PortugalLong-Term Safety and Efficacy Study of Eplontersen in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
PortugalThe busiest research cities in the country
What the registry says about research in the country
Innovative trials focusing on head and neck cancer treatments and advanced lung cancer therapies, combining novel approaches with standard treatments.
Studies evaluate targeted therapies and immunotherapy combinations to improve patient outcomes.
Comprehensive research into various autoimmune disorders, with particular focus on psoriasis and systemic sclerosis treatments.
Testing innovative biological therapies and novel treatment approaches.
Studies examining treatments for Dravet Syndrome and Alzheimer's-related psychosis, focusing on improving patient quality of life.
Investigating new therapeutic options for challenging neurological conditions.
Research targeting Crohn's Disease and various inflammatory conditions, evaluating new treatment combinations.
Testing advanced therapies for chronic inflammatory conditions.
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