Pharmaceutical Technology

JUNE 23, 2023

Sarepta will now need to complete a confirmatory trial to prove the therapy improves physical function and mobility.

Gene Therapy 130

Gene Therapy FDA Approval Gene Trials 130

Bio Pharma Dive

FEBRUARY 25, 2021

Amondys 45 is now cleared for the estimated 8% of Duchenne patients eligible for treatment. Like other Sarepta drugs, it comes with a high price tag and its clinical benefits haven't been confirmed.

FDA Approval 275

FDA Approval Drugs 275

XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). million price tag of Elevidys, a one-time gene therapy. In a media call, Sarepta CEO Douglas Ingram revealed the $3.2

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

XTalks

FEBRUARY 27, 2024

Technologies like CRISPR-Cas9 have revolutionized the potential for gene editing, offering hope for curative treatments for conditions like Duchenne muscular dystrophy (DMD) and certain types of inherited blindness. Sarepta hopes to clarify Elvidys’ effectiveness in older children in a confirmatory trial.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

FDA Law Blog

JUNE 27, 2023

Duchenne, Friederichs’s Ataxia, Sickle Cell Disease, Chagas, ALS). In addition to Mr. Karst, ten other HP&M Directors were tagged as “Recommended” by WWL: Life Sciences 2023: Ricardo Carvajal Robert A. Dormer Jeffrey N. Gibbs Paul M. Hyman Alan M. Kirschenbaum Allyson B. Mullen Frank J. Sasinowski Jeffrey K.

Life Science 81

Life Science FDA Approval Drugs Development 81

XTalks

FEBRUARY 11, 2021

They conducted two experiments, one with radioactive phosphorous to label DNA, and one with radioactive sulfur to tag protein (DNA does not contain sulfur and protein does not contain phosphate groups). Through the radioactive labels, they were able to track which of the two, DNA or protein, was injected into the bacteria.

Genetics 119

Genetics DNA Genome Genomics 119