Phase II
St. Jude Children’s Research Hospital published data from its Phase I/II clinical trial of its gene therapy for SXCID or SCID-X1, also dubbed “bubble boy disease.” The research was published in the New England Journal of Medicine.
Pharma and biotechs from across the globe share news, pipeline updates, with stories from Vetter, Atomwise, Tessa, GenSight, and more.
April 17, 2019 is the 29th World Hemophilia Day, a designated day to create awareness of the disease. Hemophilia is one of a number of bleeding disorders. The blood of people with hemophilia does not clot normally as the result of insufficient clotting factor. As a result, they can bleed for a longer time.
Janssen Pharmaceuticals presented data that showed its RNAi therapeutic candidate exhibited robust effects on the liver disease that takes the lives of more than 900,000 people annually.
The life science industry is continually making advancements in every area! We want to know what cool science innovations are currently in progress and the professionals behind them.
Axsome Therapeutics, based in New York City, announced that its AXS-05 met its primary endpoint for smoking cessation treatment. The drug significantly reduced daily smoking compared to patients on bupropion.
New data also suggest an improvement in pruritus – a major symptom of PBC – with a median change in VAS of -24% (80mg) and -49% (120mg) vs. -7% (placebo)
Gyroscope Therapeutics, headquartered in Stevenage, UK, is merging with Ambler, Pennsylvania-based Orbit Biomedical. They will operate under the Gyroscope name and focus on gene therapies for diseases of the eye.
As the International Liver Conference continues to unfold in Vienna, multiple companies are presenting data on potential treatments that can address numerous needs. BioSpace takes a look at some of the data.
April 11 is World Parkinson’s Day, designed to raise awareness of Parkinson’s disease. Parkinson’s is a progressive disease of the nervous system that affects movement.
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