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Innovation Observatory > Reports > Neurology and Neurosurgery

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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’).

Drugs

November 2017

Ponesimod for Relapsing-remitting Multiple Sclerosis

Ponesimod is a drug which works by blocking the signals which allow the body’s immune cells to travel to and damage the nerve cells. By preventing the immune cells from damaging the nerves, it is thought this drug will stop the damage which causes MS ‘relapses’. In clinical trials it has been shown that ponesimod …

Drugs

November 2017

Clostridium botulinum neurotoxin A (Xeomin) for lower limb spasticity due to cerebral palsy – first line

Botulinum toxin type A is a drug that is already in use in the UK for the treatment of a number of disorders including spastic muscular and movement conditions. It acts by selectively blocking the
release of a type of enzyme (acetylcholine) involved in spasticity in the muscle. It is administered directly into the affected muscle, providing a temporary reduction in muscular activity in the injected muscles therefore relieving spasticity.

Drugs

October 2017

Galcanezumab for the prophylaxis of episodic and chronic migraine

Galcanezumab is being developed as one of a class of specific anti-migraine preventative drugs. By stopping a very specific protein in the brain and nervous system, galcanezumab reduces migraine attacks. Additionally as it is given once-a-month as treatment, it avoids the need to take numerous pills per day. If marketed, galcanezumab may be more preferable …

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