M281 treatment prevents human NAb-mediated reduction of liver-targeted gene delivery after intravenous AAV administration in humanized FcRn transgenic mice. (A) Scheme of the in-mouse study assessing M281 effects on hIgG and anti-AAV NAb levels and subsequent systemic...
A new research paper titled “AAV1.NT-3 gene therapy prevents age-related sarcopenia” has been published in Aging. Sarcopenia is progressive loss of muscle mass and strength occurring during normal aging with significant consequences on the quality of life...
Remedium Bio today announced the publication of pre-clinical data demonstrating the safety and chondrogenic activity of RMD1101, its innovative gene therapy for Osteoarthritis. These data are the first to be published from Remedium’s ongoing research...
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that it received notice this afternoon from the U.S. Food and Drug Administration (FDA) that the agency has extended...
February 28 is Rare Disease Day, which looks to raise awareness of rare diseases and bring about change for patients and families living with them. The event also brings attention to the need for improved access to diagnosis, as well as the need to develop more...
