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Summit Therapeutics PLC (NASDAQ: SMMT LON:SUMM) has presented first clinical data for an upgraded version of ezutromid, its lead utrophin modulator, at the European Paediatric Neurology Society Congress in Lyon. Summit wants to improve absorption of ezutromid in patients with duchenne muscular dystrophy (DMD) through a modified diet and new formulation. "Our Phase 1 clinical development programme has identified ways to improve the pharmacokinetic profile of ezutromid. Through both formulation development and dietary advice, ezutromid achieves exposures which we believe have the potential to sustain utrophin production in patients with DMD," said David Roblin, Summit’s president of R&D. "We are now utilising both methods in our ongoing Phase 2 clinical trial, PhaseOut DMD, where we have the chance to see the impact of ezutromid drug exposure on pharmacology, safety and efficacy with longer term dosing of patients with DMD." Duchenne Muscular Dystrophy, or DMD, is one of the most common, fatal genetic disorders diagnosed in children around the world. It predominantly affects boys and it results in the progressive wasting of muscles throughout the body. The disease has an estimated incidence of 1 in 5,000 and a patient population in the developed world of around 50,000. Patients typically don’t live beyond their late 20s. It is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. Utrophin protein is functionally and structurally similar to dystrophin and in preclinical studies the continued expression of utrophin had a meaningful, positive effect on muscle performance.
Original Article: Summit Therapeutics presents first data from reformulated DMD treatmentNEXT ARTICLE
Neurology - Central Nervous System (CNS)
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Muscular dystrophy is a group of degenerative inherited disorders causing muscle weakness and loss of muscle tissue. The different types are Becker muscular dystrophy, Duchenne muscular dystrophy, Emery-Dreifuss muscular dystrophy, Facioscapulohumeral mu...
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