Neurologic scores stabilized, seizures diminished with tailored antisense oligonucleotide therapy The researchers who developed a personalized antisense oligonucleotide (ASO) for a little girl with a form of Batten disease — all in record time — have detailed their case in the New England Journal of Medicine. Timothy Yu, MD, PhD, of Boston Children’s Hospital, and colleagues explained how
Subretinal administration of the gene therapy AAV2/5-OPTIRPE65 showed improvements in vision-guided mobility, retinal sensitivity, and foveal-driven visual function for patients with RPE65-associated retinal dystrophy, according to findings from a phase 1/2 study presented at the 2019 American Academy of Ophthalmology Annual Meeting. In the early-stage study, there was a significant change in the ability to complete a
Analytics firm Elsevier has teamed up with non-profit phactMI to develop a new solution for providing scientifically accurate information to healthcare professionals. The deal will leverage Elsevier’s Entellect life sciences platform to create a semantic search portal. Scheduled to launch in the fourth quarter of this year, the new solution will facilitate rapid access to
Source: H. Lee Cancer Center & Research Institute Patients with advanced melanoma have been able to live longer because of several newly approved targeted treatment options, including BRAF and MEK inhibitors. However, patients will often have different responses to the same treatment due to genetic variability. Melanoma varies from patient to patient, but genetic variability is
The country’s health department is now sharing study results on new drugs and treatments online. Transparency advocates want the U.S. to do the same. Health Canada has posted safety and efficacy data online for four newly approved drugs; it plans to release reports for another 13 drugs and three medical devices approved or rejected since March. Source
The number of cell and gene therapy clinical trials in the UK continues to increase with 85 ongoing trials at present, representing a 37% increase over last year. For the first time the majority of trials have commercial sponsors, many of whom are non-UK companies. There has also been an increase in the proportion of
Report urges researchers to look outside the petri dish and into the body to study T cell metabolism Source http://www.vai.org/ For years, scientists have used cells grown in petri dishes to study the metabolic processes that fuel the immune system. But a new report in Immunity suggests looking outside the dish and into living organisms gives a
Source https://www.mintz.com/insights-center/viewpoints/2791/2019-10-fda-updates-digital-health-guidances-align-21st-century On September 26, 2019, FDA released a suite of revised digital health guidances, which includes the following: Changes to Existing Medical Software Policies Resulting from Section 3060 of the 21st Century Cures Act (“Section 3060 Changes Guidance”) – Final Policy for Device Software Functions and Mobile Medical Applications (“MMA Guidance”) – Final General Wellness: Policy for
Source http://www.liebertpub.com/ The CRISPR Journal announces the publication of its October 2019 Special Issue on The Ethics of Human Genome Editing. The Journal is led by Editor-in-Chief Rodolphe Barrangou, PhD (North Carolina State University) and Executive Editor Dr. Kevin Davies. For full-text copies of articles or to arrange interviews with Dr. Barrangou, Dr. Davies, authors, or
By Annie Hubert, Senior Director, European Public Policy at Alliance for Regenerative Medicine ATMPs are varied and complex, ranging from potential one-off curative treatments to treatments tailored to a specific individual. As such, they differ significantly from ‘traditional’ medicinal treatments, something which has been recognised by regulators in Europe: the European Parliament introduced ATMPs as a
