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Branaplam has been demonstrated to decrease levels of mutant huntingtin protein in preclinical models, said Novartis. Based on the results, Novartis aims to begin a development programme for

Under the terms of the agreement EffRx has received the exclusive rights to register and commercialize Alkindi in Switzerland. Alkindi is the first preparation of hydrocortisone specifically designed

NantKwest and ImmunityBio’s hAd5-Covid-19 is a novel Covid-19 vaccine candidate designed to target the inner nucleocapsid (N) and the outer spike (S) protein, as well as activate both

While up to 30% of all patients with NSCLC may be diagnosed early enough to have potentially curative surgery, disease recurrence is still common in early-stage disease and

Xeris’ XP-0863 was previously granted orphan designations both for the treatment of acute repetitive seizures and for the treatment of Dravet Syndrome. The FDA’s Fast Track designation highlights

PMM is a complex mitochondrial disease in which genetic mutations mainly impair the function of mitochondria that may result in reduced muscle function, reduced endurance to exercise, increased

With a combined area of 8,700m² in the Stellar 32 campus within Geneva’s ZIPLO, the newly expanded facility will co-locate primary European operations of both companies. KBI Biopharma’s

ROR1 is a cell-surface protein that is expressed during embryofetal development but disappears before birth and is usually not expressed on normal cells in children or adults. However,

UCD is a rare and life-threatening condition caused by an inherited inborn error of metabolism. Current treatment options are associated with poor outcomes. “The rare pediatric disease designation