Archives: 2019-09-22

Saving lives faster: Monash develops world-first laser incubator for blood

Monash University researchers, along with industry partner Haemokinesis, have developed the world’s first blood incubator using laser technology. This technology can slash blood incubation time to just 40 seconds, compared to the current industry gold standard of five minutes. The study shows that laser incubation can improve pre-transfusion testing for millions of patients undergoing blood

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In Mice: Transplanted Brain Stem Cells Survive Without Anti-Rejection Drugs

By exploiting a feature of the immune system, researchers open the door for stem cell transplants to repair the brain. In experiments in mice, Johns Hopkins Medicine researchers say they have developed a way to successfully transplant certain protective brain cells without the need for lifelong anti-rejection drugs. A report on the research, published Sept. 16 in

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World’s First Gene Therapy for Glycogen Storage Disease Produces Remarkable Results

At the Association for Glycogen Storage Disease’s 41st Annual Conference, Dr. David Weinstein of UConn School of Medicine and Connecticut Children’s presented his groundbreaking, one-year clinical trial results for the novel gene therapy treatment for glycogen storage disease (GSD). The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously

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Bluebird bio Presents Updated Data from Phase 2/3 Clinical Study of Lenti-D™ Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress

Long-Term Follow-up Data Show That the 88% of Patients Treated in the Starbeam Study (ALD-102) Were Free of Major Functional Disabilities (MFDs) at Two Years, and Continued to Remain MFD-Free at up to Five Years of Follow-up  bluebird bio, Inc. (Nasdaq: BLUE) today announced updated results from the clinical development program for its investigational Lenti-D™ gene

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Suvodirsen Receives FDA’s Fast Track Status to Treat Duchenne Muscular Dystrophy

The U.S. Food and Drug Administration (FDA) has granted fast track designation to suvodirsen as a treatment for people with Duchenne muscular dystrophy (DMD). The agency’s decision — which will expedite the review process for suvodirsen — was based on experimental and preclinical data demonstrating the treatment’s potential therapeutic activity. Suvodirsen already had been granted orphan drug designation for the treatment of DMD

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FDA Grants Orphan Drug Designation to Reneo Pharmaceuticals for REN001 for Treatment of Fatty Acid Oxidation Disorders

 Reneo Pharmaceuticals, a clinical stage pharmaceutical company, announced today that the U.S. Food and Drug Administration  (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to the company’s lead drug candidate, REN001, for the treatment of fatty acid oxidation disorders (FAOD). “There is a pressing need for effective treatments for patients suffering from

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Medical Board of California meeting on stem cell clinics emphasized the rigorous stem cell-related clinic trials ongoing and in development and the risks coming from deviating from standard clinical trial and GMP practices.

Source The Niche Knoepfler Lab Stem Cell Blog This afternoon Dr. Knoepfler attended and gave public comment at the Medical Board of California stem cell task force meeting on unproven stem cell clinics. There were about 40 people attending. In part this meeting is a consequence of the national governing organization of state medical boards, the Federation of

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