XTalks

DECEMBER 29, 2022

The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

SEPTEMBER 12, 2022

Sanofi picked up its first Xenpozyme approval for the treatment of Niemann-Pick from Japanese regulators in March followed by an approval from European officials in May. The FDA approval was based on data from the randomized, double-blind, placebo-controlled ASCEND study involving 31 adult patients.

FDA Approval 98

FDA Approval Genetics Trials Regulation 98

XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. In March, the US Food and Drug Administration (FDA) approved Daybue (trofinetide) , the first and only treatment for Rett syndrome. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

XTalks

AUGUST 6, 2020

The US Food and Drug Administration (FDA) has approved the use of an oral cannabidiol (CBD) solution called Epidiolex for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients aged one year and older. Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Surrogate endpoints were used as the basis for approval of 45 percent of new drugs reviewed by the FDA between 2010 and 2012. Nizoral is prescribed off-label, while the others are FDA approved.

Trials 98

Trials Gene Hormones Clinical Trials 98