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Innovation Observatory > Reports > Neurology and Neurosurgery

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Drugs

August 2018

Inebilizumab for Neuromyelitis optica

Inebilizumab is a humanised monoclonal antibody that is in clinical development for reducing the risk of an attack in patients with Neuromyelitis Optica Spectrum Disorders (NMOSD). NMOSD, previously known as NMO and as Devic’s disease, is a rare, disabling autoimmune disease of the central nervous system. It predominantly affects the optic nerve and spinal cord, …

Drugs

June 2018

Sarizotan for Respiratory Symptoms Associated Rett Syndrome

Sarizotan is an oral medicinal product that is being developed for the treatment of respiratory symptoms associated with Rett syndrome. Sarizotan works by binding to serotonin and dopamine receptors. By stimulating serotonin and dopamine receptors, sarizotan replaces the effect of some of the missing serotonin in the brain and spinal cord. This is expected to help restore normal breathing rhythm in patients with Rett syndrome. Currently, there is no cure for Rett syndrome and treatment focuses on managing the symptoms. If licensed, sarizotan could become the first therapy approved for treatment of Rett Syndrome patients.

Drugs

June 2018

Cenobamate for Partial Focal Epilepsy – Adjunctive Therapy

Cenobamate is a medicinal product that is being developed as a therapy for patients with partial focal epilepsy that would be taken in addition to other anti-epileptic medicine (adjunctive therapy). It is given as capsules. Cenobamate is considered a new generation antiepileptic therapy and clinical trials have shown that it may be more effective and safer than existing drugs. If licensed, cenobamate will offer a new adjunctive treatment option for patients with partial focal epilepsy.

Drugs

April 2018

AVXS-101 for spinal muscular atrophy

AVXS-101 is a gene replacement therapy, made of a virus that has been modified to contain the primary gene for the SMN protein, which is lacking (or mutated) in patients with SMA. When injected into the patient, the virus is expected to carry the gene into the nerve cells, enabling them to start producing sufficient amounts of SMN. This is expected to improve the survival and function of the motor neurons, and so preserve muscle function. AVXS-101 is thought to address the root cause of SMA and therefore, if licensed, may offer an additional treatment option for patients with spinal muscular atrophy.

Drugs

February 2018

Pentetrazol for Idiopathic Hypersomnia and Narcolepsy Type 2

Pentetrazol is a medicinal product that is being developed for the treatment of IH and narcolepsy type 2. Pentetrazol is administered orally and it acts by blocking the effect of a chemical substance called Gamma-Aminobutyric Acid (GABA). GABA is thought to play a role in promoting sleeping and is believed to be elevated in people with IH. If licensed, Pentetrazol will offer a new treatment option for patients with IH or narcolepsy type 2.

Drugs

January 2018

Perampanel (Fycompa) for Paediatric Epilepsy

Epilepsy is a condition in which the brain is affected by abnormal discharge of electrical activity, causing seizures (fits). There are several different types of epileptic seizures, depending on what part of the brain they start in and which part they affect. Epilepsy can start in any age, but is most common in either childhood …

Drugs

January 2018

PXT3003 for Charcot-Marie-Tooth Disease Type 1A

Charcot-Marie-Tooth disease type 1A (CMT1A) is an inherited genetic disease in which the motor and/or sensory peripheral nerves are affected, resulting in muscle weakness and wasting, as well as sensory loss.

Drugs

January 2018

Ronopterin for Moderate to Severe Traumatic Brain Injury

Ronopterin is being developed as a continuous infusion that can be used individually or in combination with the current standard treatments for traumatic brain injury. The current medications available are only used to treat the symptoms of traumatic brain injury.

Drugs

December 2017

Dabigatran Etexilate (Pradaxa) for Secondary Stroke Prevention in Patients with Embolic Stroke of Undetermined Source – First Line

Dabigatran etexilate is an oral drug that acts by preventing clots from forming and blocking blood vessels. It is already available for the treatment and prevention of stroke and other types of blood clots affecting blood vessels and organs in the body. By preventing clots from forming, it can prevent future embolic strokes.

Drugs

December 2017

Cannabidiol (Epidiolex®) for Tuberous Sclerosis Complex – add-on therapy

Cannabidiol is a medicine that is being developed for the treatment of TSC patients who experience inadequately-controlled seizures. It is being developed as an oral medicine to be added to existing treatment options for TSC (‘add-on therapy’).

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