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 and gene therapy answers: Overcoming challenges for rare disease in pediatrics (part two)
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, Labcorp cell and gene therapy leadership shared their insights on strategies for overcoming key challenges in rare disease studies in pediatric populations along with their …
Cell and Gene Therapy Answers: Advancing treatments for pediatric patients with rare diseases
Your source for answers to the complex challenges of cell and gene therapy development. Labcorp’s cell and gene therapy scientific leaders shared their insights on the factors that make rare and pediatric diseases a good fit for cell and gene therapy, along with the key challenges and best practices in …
The Evolution of Open-Label Extensions and Rare Disease Studies
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 …
Making Sense of Antisense Oligonucleotide-Based Therapies in Muscular Dystrophies
Good news for the Duchenne Muscular Dystrophy (DMD) community. On June 8th, BioMarin announced the filing of a Marketing Authorization Application to the European Medicines Agency for Drisapersen, an antisense-mediated exon 51-skipping compound able to target the most prevalent genetic mutations responsible for the lack of production of functional dystrophin. …