Navigating the Complexities of Age-Appropriate Dosing: The Role of Newborn Screening in Guiding Treatment Decisions

Time: 1:00 pm
day: Pre-Conference Day


The earlier a patient can be treated with a gene therapy, the more effective the gene therapy is likely to be. Join this workshop to dive into the recent success of newborn screening for DMD and the positive impact this can have on age of dosing guidelines for muscular gene therapies.

Attend this workshop to learn about:

  • Discussing the importance of reliable newborn screening for degenerative muscular disorders to help avoid the diagnostic odyssey that families may go through
  • Delving into the requirements for the condition to be added the recommended uniform screening panel and why there maybe adversities to this
  • Exploring the optimal age of dosing if a patient has been diagnosed with DMD at birth
  • Navigating the challenges surrounding pre-symptomatic dosing
  • Assessing the potential for increased efficacy related to earlier dosing
  • Latest updates on the progression of studies regarding newborn screening for DMD