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.
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.
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 …
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.
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.
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 …
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.
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 …