LEXINGTON, Mass. – LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today announced results from a retrospective study of the disease course in pediatric patients living with severe methylmalonic acidemia (MMA) caused...
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North Bethesda, Maryland – Long-acting antiretroviral therapy (ART) with cabotegravir and rilpivirine was superior in suppressing HIV replication compared to daily oral ART in people who had been unable to maintain viral suppression through an oral daily regimen, according to interim data from a randomized trial. Upon review of these...
Nijmegen, Netherlands – The cause of rare diseases is increasingly being detected through genome sequencing, which involves reading the entire human DNA by first breaking it into small pieces—short reads. Christian Gilissen, Lisenka Vissers, and colleagues found that a new technique using long reads is even more effective at detecting...
Nashville, Tennessee – A 10-year follow up study of nearly 2,500 U.S. men who received prostate cancer treatment will help inform decision making in terms of treatments and side effects for a diverse population. The CEASAR (Comparative Effectiveness Analysis of Surgery and Radiation for Localized Prostate Cancer) study, coordinated by...
Cleveland, Ohio – Older patients with Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) who were not good candidates for the standard treatment of intensive chemotherapy had a median overall survival (OS) of 6.5 years on an alternate regimen of dasatinib and blinatumomab. These long-term results from the S1318 clinical trial...
London, UK – A highly sensitive bone marrow test could double survival rates for some groups of younger adults with Acute Myeloid Leukaemia (AML) by helping doctors identify if they might relapse up to three months earlier. The patient-specific molecular test can detect low levels of leukaemia cells in the...
Tokyo, Japan – Researchers from Tokyo Metropolitan University have studied pancreatic islet cell loss in people with no previous pancreatic problems. They identified key trends in the types of cells lost due to islet cell loss in different age groups and sexes, finding that ICL in the elderly population was...
Davis, California – New UC Davis research shows that men with an FMR1 premutation who experienced reduced executive function were at higher likelihood of developing fragile X-associated tremor/ataxia syndrome (FXTAS). FXTAS is a progressive genetic condition that causes movement issues and cognitive decline. These findings, published in the journal Movement...
Portland, OR – New research from Oregon Health & Science University suggests that people with multiple sclerosis, or MS, could benefit from a low-fat diet to improve the fatigue that’s a debilitating, and often-underappreciated, symptom of the condition. The study, published online Wednesday in the Multiple Sclerosis Journal, is the latest in...
Saint Louis, Missouri – In Alzheimer’s disease and related dementias, cognitive decline is driven by the overaccumulation of a normal brain protein known as tau. Wherever tau builds up, nearby brain tissue starts to degenerate and die. Now, researchers at Washington University School of Medicine in St. Louis have found...