Everyone in this world has needs but not all people have the same needs. Tabia McKinzie wants to raise awareness for people who have what most would call “special needs.” This has been brought to the forefront for McKinzie because of her daughter, Nandi. Nandi has Turner syndrome, a rare...
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SAN DIEGO — Locanabio, Inc., a genetic medicines company developing RNA-targeted therapeutics for patients with rare genetic neuromuscular and neurodegenerative diseases, announced that CureDuchenne Ventures, the strategic investment arm of CureDuchenne, has made an equity investment in Locanabio. This investment will support the development of the company’s snRNA-mediated exon skipping...
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...
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 an update on screening and enrollment activities for the SUNRISE Phase I/II clinical trial of LB-001, the company’s investigational treatment for...
LEXINGTON, Mass. – LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood, today announced that the first patient has been dosed with LB-001, the Company’s investigational single-administration gene editing therapy based on its proprietary...
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 the promotion of Daniel Gruskin, MD, as chief medical officer. Dr. Gruskin most recently served as senior vice president and head of clinical...
LEXINGTON – LogicBio Therapeutics, Inc. (Nasdaq: LOGC), a clinical-stage genetic medicine company, today provided an update on the LB-001 clinical development program. The U.S. Food and Drug Administration (FDA) has notified the company that its Phase 1/2 SUNRISE clinical trial of LB-001 in pediatric patients with methylmalonic acidemia (MMA) has...
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 the appointment of Andrea Paul as general counsel and corporate secretary and Janice Olson as senior vice president of...
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...