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5/01/2020

Spotlight on the News: COVID-19, Albion Coll., ACLU MI.; James Wilson, Mauri Ditzler & Dan Korobkin

On Sunday, May 3, Spotlight on the News will focus on the race to find a vaccine to protect humans from the COVID-19 virus. Noted University of Pennsylvania scientist Dr. James M. Wilson grants Spotlight his first Detroit television interview on why his lab was picked to search for a cure. We'll also talk to Albion College President Mauri Ditzler about the school's Michigan Promise Scholarship Program and Albion's connection to Dr. Wilson. And how has the ACLU of Michigan been impacted by the Coronavirus? Legal Director Dan Korobkin will tell us.

2/28/2020

Rare Disease Day 2020

Tomorrow we celebrate Rare Disease Day to raise awareness for the 300 million individuals worldwide who live with rare diseases.

2/27/2020

Passage Bio Announces Pricing of Initial Public Offering

2/12/2020

CRISPR “Minigene” Approach Stops Genetic Liver Disease in Mice

A new CRISPR gene-editing technique prevented a genetic liver disease known to be driven by hundreds of different mutations and improved clinical symptoms in mice, Penn Medicine researchers reported in new proof-of-concept study published online in Science Advances. The findings suggest a promising CRISPR tool that could potentially treat patients with a rare metabolic urea-cycle disorder caused by a deficiency the enzyme, ornithine transcarbamylase (OTC), as well as other hereditary diseases triggered by different mutations on the same gene.

1/28/2020

FDA Continues Strong Support of Innovation in Development of Gene Therapy Products

This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient’s cells. The agency anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area. The FDA believes this will provide patients and providers with increased therapeutic choices.

1/27/2020

Rare Disease CDKL5 Deficiency Disorder Granted WHO Disease Classification

The rare genetic disease CDKL5 Deficiency Disorder (CDD) has been designated with a new disease code in the International Classification of Diseases (ICD), the medical classification list from the World Health Organization (WHO). The CDKL5 Deficiency Disorder diagnostic code will be incorporated in the October 1, 2020 classification revision.