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Nusinersen

Index Nusinersen

Nusinersen, marketed as Spinraza, is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. [1]

33 relations: Alternative splicing, Antisense therapy, Biogen, Blood plasma, Central nervous system, Cerebrospinal fluid, Cision, Cold Spring Harbor Laboratory, Cytochrome P450, Drug discovery, European Medicines Agency, European Union, Intrathecal administration, Ionis Pharmaceuticals, List price, Marketing authorization, Mutation, New Drug Application, Oligonucleotide, Orphan drug, Priority review, Pulmonary aspiration, Scoliosis, SMN1, SMN2, Spinal muscular atrophy, Survival of motor neuron, The New York Times, Thiophosphate, Thrombocytopenia, Unified atomic mass unit, United States, University of Massachusetts Medical School.

Alternative splicing

Alternative splicing, or differential splicing, is a regulated process during gene expression that results in a single gene coding for multiple proteins.

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Antisense therapy

Antisense therapy is a form of treatment for genetic disorders or infections.

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Biogen

Biogen, Inc. (previously known as Biogen Idec) is an American multinational biotechnology company based in Cambridge, Massachusetts, specializing in the discovery, development, and delivery of therapies for the treatment of neurodegenerative, hematologic, and autoimmune diseases to patients worldwide.

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Blood plasma

Blood plasma is a yellowish coloured liquid component of blood that normally holds the blood cells in whole blood in suspension; this makes plasma the extracellular matrix of blood cells.

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Central nervous system

The central nervous system (CNS) is the part of the nervous system consisting of the brain and spinal cord.

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Cerebrospinal fluid

Cerebrospinal fluid (CSF) is a clear, colorless body fluid found in the brain and spinal cord.

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Cision

Cision Ltd. is a public relations and earned media software company and services provider.

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Cold Spring Harbor Laboratory

Cold Spring Harbor Laboratory (CSHL) is a private, non-profit institution with research programs focusing on cancer, neuroscience, plant genetics, genomics, and quantitative biology.

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Cytochrome P450

Cytochromes P450 (CYPs) are proteins of the superfamily containing heme as a cofactor and, therefore, are hemoproteins.

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Drug discovery

In the fields of medicine, biotechnology and pharmacology, drug discovery is the process by which new candidate medications are discovered.

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European Medicines Agency

The European Medicines Agency (EMA) is a European Union agency for the evaluation of medicinal products.

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European Union

The European Union (EU) is a political and economic union of EUnum member states that are located primarily in Europe.

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Intrathecal administration

Intrathecal administration is a route of administration for drugs via an injection into the spinal canal, or into the subarachnoid space so that it reaches the cerebrospinal fluid (CSF) and is useful in spinal anaesthesia, chemotherapy, or pain management applications.

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Ionis Pharmaceuticals

Ionis Pharmaceuticals (known as Isis Pharmaceuticals until December 2015) is a publicly traded pharmaceutical company based in Carlsbad, California, United States.

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List price

The list price, also known as the manufacturer's suggested retail price (MSRP), or the recommended retail price (RRP), or the suggested retail price (SRP), of a product is the price at which the manufacturer recommends that the retailer sell the product.

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Marketing authorization

Process of reviewing and assessing the dossier to support a medicinal product in view of its marketing (also called licensing, registration, approval, etc.), finalized by granting of a document also called marketing authorization (MA) (equivalent: product license).

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Mutation

In biology, a mutation is the permanent alteration of the nucleotide sequence of the genome of an organism, virus, or extrachromosomal DNA or other genetic elements.

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New Drug Application

The Food and Drug Administration's New Drug Application (NDA) is the vehicle in the United States through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing.

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Oligonucleotide

Oligonucleotides are short DNA or RNA molecules, oligomers, that have a wide range of applications in genetic testing, research, and forensics.

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Orphan drug

An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.

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Priority review

Priority review is a mechanism used by the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease.

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Pulmonary aspiration

Pulmonary aspiration is the entry of material (such as pharyngeal secretions, food or drink, or stomach contents) from the oropharynx or gastrointestinal tract into the larynx (voice box) and lower respiratory tract (the portions of the respiratory system from the trachea—i.e., windpipe—to the lungs).

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Scoliosis

Scoliosis is a medical condition in which a person's spine has a sideways curve.

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SMN1

Survival of motor neuron 1 (SMN1), also known as component of gems 1 or GEMIN1, is a gene that encodes the SMN protein in humans.

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SMN2

Survival of motor neuron 2 (SMN2) is a gene that encodes the SMN protein (full and truncated) in humans.

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Spinal muscular atrophy

Spinal muscular atrophy (SMA) is a rare neuromuscular disorder characterised by loss of motor neurons and progressive muscle wasting, often leading to early death.

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Survival of motor neuron

Survival of motor neuron or survival motor neuron (SMN) is a protein that in humans is encoded by the SMN1 and SMN2 genes.

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The New York Times

The New York Times (sometimes abbreviated as The NYT or The Times) is an American newspaper based in New York City with worldwide influence and readership.

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Thiophosphate

Thiophosphates (or phosphorothioates, PS) are chemical compounds and anions with the general chemical formula PS4−xO (x.

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Thrombocytopenia

Thrombocytopenia is a condition characterized by abnormally low levels of thrombocytes, also known as platelets, in the blood.

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Unified atomic mass unit

The unified atomic mass unit or dalton (symbol: u, or Da) is a standard unit of mass that quantifies mass on an atomic or molecular scale (atomic mass).

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United States

The United States of America (USA), commonly known as the United States (U.S.) or America, is a federal republic composed of 50 states, a federal district, five major self-governing territories, and various possessions.

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University of Massachusetts Medical School

The University of Massachusetts Medical School (UMMS) is one of five campuses of the University of Massachusetts (UMass) system.

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Redirects here:

IONIS-SMNRx, ISIS-SMNRx, SMNRx, Spinraza.

References

[1] https://en.wikipedia.org/wiki/Nusinersen

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