XTalks

APRIL 24, 2023

Melanie Blank, clinical team leader for General Medicine Branch 1 at the US Food and Drug Administration’s (FDA) Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), the agency is seeing one or two new applications coming in every week for new gene therapies for different diseases. Reference: Chung DC, et al.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

MARCH 3, 2021

Molybdopterins are a class of cofactors found in most molybdenum-containing and all tungsten-containing enzymes. The efficacy of Nulibry in the treatment of MoCD Type A was demonstrated in data from three different clinical trials that was compared to data from a natural history study.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

SEPTEMBER 15, 2020

A vaccine developed by Swedish company Diamyd Medical has demonstrated significant treatment efficacy in a predefined genetic subgroup of individuals with type 1 diabetes in a Phase IIb clinical trial. So, this really validates what we already knew and now we have prospective results supporting that [from this trial].”.

Vaccination 103

Vaccination Vaccine Insulin Trials 103

XTalks

APRIL 15, 2021

Late-stage clinical trial failures are largely to blame for the lack of effective treatment options for patients with HFpEF, and poor five-year survival rates make this an area of great unmet need. A lack of efficacy is the greatest reason for clinical trial failures, especially in Phase II and Phase III,” says Dr. Tyl.

Development 98

Development Drugs Genome Genomics 98

XTalks

FEBRUARY 4, 2021

Through these collaborations, cohort research dataset sharing programs allow for whole-genome sequence and genotype data to be made available for independent research. Now, if all you’ve already got is a bioresource of 20,000 people that have already been genotyped, you can choose those from that sample and focus your effort.”.

Research 98

Research Biobanking Drugs Genome 98