Grand Rounds: Targeted Therapies in Skeletal Dysplasias
03-21-2025
By Jason Laufman, MD, FACMGG , Geneticist, Akron Children's, Akron, OH
Objectives (Educational Content) :
1. Understand orphan drugs and their utility. 2. Learn about new therapies for rare disorders. 3. Understand when to refer rate disease to genetics.
Target Audience:
General pediatricians, family physicians, nurse practitioners, physician assistants, social workers, psychologists, and nurses.
Identified Gap:
Despite advancements in genetic research, there is a lack of widespread understanding and application of targeted therapies in the treatment of skeletal dysplasia, highlighting the need to educate healthcare providers on the history, types, and availability of these personalized therapies to improve patient outcomes.
Estimated Time to Complete the Educational Activity:
1 hour(s)
Expiration Date for CE/CME Credit:
03-20-2026
Method of Participation in the Learning Process:
The learner will view the presentation, successfully complete a post-test and complete an activity evaluation.
Evaluation Methods:
All learners must successfully complete a post-test, as well as an activity evaluation, to claim CE/CME credit.
Disclosure:
The following presenter has indicated that a financial relationship with an ineligible company: Biomarin Pharmaceuticals Jason Laugman, MD. The following planning committee member has indicated that a financial relationship with an ineligible company: All relationships have been mitigated. The remaining planning committee/CME committee members have indicated that they have no financial relationships with any ineligible companies.
Accreditation Statement:
Akron Children’s Hospital is accredited by the Ohio State Medical Association to provide continuing medical education for physicians.
Akron Children’s Hospital designates this enduring material activity for a maximum of 1.0 AMA PRA Category 1 Credit TM. Physicians should only claim the credit commensurate with the extent of their participation in the activity.
Bibliography:
1. Sabir, A.H., Cole, T. The evolving therapeutic landscape of genetic skeletal disorders. Orphanet J Rare Dis 14, 300 (2019). https://doi.org/10.1186/s13023-019-1222-2
2. Death but one unintended consequence of gene-therapy trial. Barbara Sibbald. CMAJ May 2001, 164 (11) 1612;
3. DOI: 10.1056/NEJM199008303230904
