Data from EMA (European Medicines Agency) - Curated by EPG Health - Last updated 03 July 2018

Indication(s)

Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy.

Full Prescribing information

Learning Zones

An epgonline.org Learning Zone (LZ) is an area of the site dedicated to providing detailed self-directed medical education about a disease, condition or procedure.

Migraine Knowledge Centre

Migraine Knowledge Centre

The Migraine Knowledge Centre features latest research on the prevalence and impact of migraine, the proposed neurological basis of the condition (and how this is being translated into new and exciting drug therapies), as well as current patient care strategies collated from headache organisations worldwide.

Chronic Lymphocytic Leukaemia (CLL)

Chronic Lymphocytic Leukaemia (CLL)

Refine your knowledge of chronic lymphocytic leukaemia (CLL) with information on pathophysiology, diagnosis, treatment options and more

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Hyperammonaemia

Hyperammonaemia

Hyperammonaemia can result in serious neurological damage or death. Could you recognise the signs and symptoms?

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Journal articles

Cerliponase Alfa: First Global Approval.

Cerliponase alfa (Brineura™) is a recombinant human tripeptidyl peptidase-1 (TPP1) being developed by BioMarin Pharmaceutical Inc. for use in patients with neuronal ceroid lipofuscinosis type 2 (CLN2)...

Added 1 year ago

Is amyotrophic lateral sclerosis/frontotemporal dementia an autophagy disease?

Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are neurodegenerative disorders that share genetic risk factors and pathological hallmarks. Intriguingly, these shared factors result in a high rate of comorbidity of...

Added 6 months ago

Advances, challenges and future directions for stem cell therapy in amyotrophic lateral sclerosis.

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative condition where loss of motor neurons within the brain and spinal cord leads to muscle atrophy, weakness, paralysis and ultimately death...

Added 6 months ago

The heat shock response in neurons and astroglia and its role in neurodegenerative diseases.

Protein inclusions are a predominant molecular pathology found in numerous neurodegenerative diseases, including amyotrophic lateral sclerosis and Huntington's disease. Protein inclusions form in discrete areas of the brain characteristic...

Added 6 months ago

Association Between Motor Symptoms and Brain Metabolism in Early Huntington Disease.

Objective: To investigate the association between brain hypometabolism and hypermetabolism with motor scores of patients with early HD.

Added 6 months ago

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Guidelines

Spasticity in under 19s: management

This guideline covers managing spasticity and co-existing motor disorders and their early musculoskeletal complications in children and young people (from birth up to their 19th birthday) with non-progressive brain disorders.

Added 5 years ago

EFNS guidelines on the Clinical Management of Amyotrophic Lateral Sclerosis (MALS) – revised report of an EFNS task force

Objectives: To provide evidence-based or expert recommendations for the diagnosis and management of ALS based on a literature search and the consensus of an expert panel.

Added 7 years ago

Use of antibody testing in nervous system disorders

To evaluate service provision and quality assurance schemes for clinically useful autoantibody tests in neurology.

Added 7 years ago

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View all 1438 Guidelines items

More information

Category Value
Agency product number EMEA/H/C/004312
Orphan designation Yes
Date First Approved 30-05-2017
Type Medicinal product subject to restricted medical prescription
Marketing authorisation holder Biogen Idec Ltd