Rare Disease Archives - Insights From Our Labs to Yours

Rare Disease Day 2022: Supporting patients, celebrating advancements

by Labcorpupdated on Monday, November 21, 2022Tuesday, February 22, 2022

February 28 is Rare Disease Day, a day dear to the hearts of many at Labcorp. The day is observed to raise awareness and give support to the more than 300 million people worldwide living with rare diseases, as well as their family members and caretakers. At the heart of …

Cell & Gene Therapy Answers: Overcoming challenges for rare disease in pediatrics part two

by Fred Derosier, Susanne Schmidt, and Isabella Preschupdated on Monday, November 21, 2022Friday, February 18, 2022

Your source for answers to the complex challenges of cell and gene therapy development. Building on the topic of rare diseases in our earlier blog, three cell and gene therapy leaders from Labcorp Drug Development shared their insights on strategies for overcoming key challenges in rare disease studies in pediatric …

Cell & Gene Therapy Answers: Advancing treatments for pediatric patients with rare diseases

by Fred Derosier, Susanne Schmidt, and Isabella Preschupdated on Monday, November 21, 2022Thursday, February 10, 2022

Your source for answers to the complex challenges of cell and gene therapy development. We recently spoke with three cell and gene therapy leaders from Labcorp Drug Development: Fred Derosier, Vice President and General Manager for Rare Diseases in Pediatrics and Clinical Lead, Enterprise Cell and Gene Therapy; Susanne Schmidt, …

Rare diseases are not so rare

by Cindy Jackson, DO, FAAP, VP and General Manager, Rare Diseases/Orphan Drugs and Pediatricsupdated on Monday, June 7, 2021Tuesday, February 25, 2020

Some rare diseases have famous fundraising events attached to them, like the Ice Bucket Challenge for ALS or the many walks, runs, and telethons for diseases ranging from cystic fibrosis to muscular dystrophy. Other conditions, like Wilson’s Disease, a rare condition in which excess copper builds up in the body, …

Covance & FDNA to develop novel program to accelerate and improve the accuracy of rare disease patient recruitment for clinical studies

by Labcorpupdated on Monday, June 7, 2021Monday, March 25, 2019

FDNA, a digital health company at the forefront of artificial intelligence (AI), genomics and precision medicine, and Covance will work together to create a new, innovative approach to clinical study recruitment for rare diseases.

What Child Health Day means to me: Gina Calarco shares her story

by Gina Calarco, MPH, BSN, RN, CCRCupdated on Monday, June 7, 2021Tuesday, October 2, 2018

On National Child Health Day, and every day, I feel grateful for the work I do, and how it impacts the lives and health of children. I also recognize this event as an opportunity to spread knowledge about the work still remaining.

Moving the needle for rare diseases

by Cindy Jackson, DO, FAAP, VP and General Manager, Rare Diseases/Orphan Drugs and Pediatricsupdated on Monday, June 7, 2021Wednesday, February 28, 2018

Each year, we designate the last day in February as Rare Disease Day to raise awareness about the potential impact we can make for this important segment of the world’s population. From policy makers to researchers to health authorities, we all play a part in making a difference for more …

The Importance of Stakeholder Research in Rare Disease Drug Development

by Labcorpupdated on Monday, February 17, 2020Tuesday, August 23, 2016

The approval of novel orphan drug designations continues to grow, while many existing rare disease therapies are receiving approval for expanded indications. With this increase and broadening class of products, including some that target the same mutation or molecular defect, sponsors face new and significant market access challenges in securing …

Advancing Rare Disease Drug Development: Four Key Considerations for Market Access

by John D. McDermott, Jr, MBAupdated on Wednesday, May 19, 2021Monday, April 18, 2016

Our industry is witnessing increasing growth in the rare disease market, thanks to financial and regulatory incentives to develop orphan drugs. This has been good news for both sponsors and patients, but the fact remains that rare disease trials are inherently challenging to run. In addition, completing a complex study …

The Evolution of Open-Label Extensions and Rare Disease Studies

by Leone Atkinson, MD, PhDupdated on Monday, February 17, 2020Tuesday, March 22, 2016

When running early development studies, sponsors must consider whether or not to provide open-label, long-term treatment at the end of their randomized, placebo-controlled trial. In the past, sponsors have been hesitant to offer open-label extensions. But with our industry’s increasing focus on patient-centric care, sponsors are now weighing their options to incorporate more …