Tagged with NCATS

Spearheading the Charge Against Rare Disease

USA Spearheading the Charge Against Rare Disease

USA The US rare and ultra-rare disease market constitutes a cornerstone of the worldwide pharmaceutical industry, representing a full 52.3 percent of the total global market share in revenue for that therapeutic area. America also plays an outsized role in driving international medical innovation in that field – housing nearly 60…

PJ Brooks – Acting Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS)

USA PJ Brooks – Acting Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS)

USA How can rare disease innovation move faster without lowering scientific or regulatory standards? In this interview, PJ Brooks explains how the National Center for Advancing Translational Sciences is shifting rare disease development toward platform-based thinking, shared molecular pathways, and early regulatory alignment. From genome editing and gene therapy to open…

PJ Brooks – Program Officer, Division of Clinical Innovation and Office of Rare Diseases Research, NCATS, NIH, USA

USA PJ Brooks – Program Officer, Division of Clinical Innovation and Office of Rare Diseases Research, NCATS, NIH, USA

USA PJ Brooks PhD, program director in the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH) in the USA, highlights the importance of going beyond one disease at a time in clinical trials, some of the roadblocks that still remain…