November 2020
rAAVrh74.MHCK7.micro-dystrophin for Duchenne muscular dystrophy
rAAVrh74.MHCK7.micro-dystrophin is a medicinal product in clinical development for the treatment of children aged 3 months to 7 years with Duchenne muscular dystrophy (DMD). DMD is a rare progressive neuromuscular disorder caused by a gene mutation (change). DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. It affects …
November 2020
Tideglusib for congenital myotonic dystrophy
Tideglusib is being development for the treatment of congenital myotonic dystrophy type 1 (CMD1). CMD1 is a form of myotonic dystrophy type 1 (DM1), a rare, genetically determined neuromuscular disorder. CMD1 begins at or around the time of birth and is characterised by severe muscle weakness, cognitive impairment and other developmental abnormalities. The condition usually occurs when the mother already has DM1 and then it is passed on to her child in a more severe form. CMD1 is typically associated with significant medical morbidity and early death. No specific treatment is currently on offer, although supportive care to manage symptoms is available.
September 2020
Istradefylline for patients with Parkinson’s disease experiencing end of dose fluctuations
Istradefylline is a selective adenosine A2A receptor inhibitor; these receptors are found in the region of the brain that suffers degeneration in PD. The treatment works by increasing the levels of dopamine in the substantia nigra. Current treatment for PD is associated with adverse events. Several phase III clinical trials have shown istradefylline, when taken orally, to be safe and effective in the treatment of PD during OFF periods. If licensed, istradefylline will provide a new therapeutic option to managing people with Parkinson’s as the first non-dopaminergic add-on treatment.
September 2020
ABBV-951 for motor fluctuations in Parkinson’s disease
ABBV-951 is administered under the skin (subcutaneous infusion) to deliver therapeutic quantities of the drugs levodopa and carbidopa. Levodopa can be converted by the body into dopamine in order to supplement the low levels of dopamine in PD patients and improve motor symptoms. Carbidopa makes more levodopa available for transport into the brain. ABBV-951 eliminates the ‘wearing off’ effect by providing a continuous infusion of levodopa into the bloodstream so there is less fluctuation in dopamine levels and improved motor control. If licenced, ABBV-951 would provide an additional treatment option for PD patients who are levodopa responsive and who have inadequately controlled motor fluctuations.
