#401 The First Precision Medicine for Achondroplasia with Dr. Ravi Savarirayan

Just a few years ago, there were no pharmacological treatments for patients with achondroplasia. Today, with multiple therapies on the market, the conversation has shifted from if we can treat to how we treat safely, consistently, and thoughtfully.

In this third and final installment of our achondroplasia series, sponsored by BioMarin, we explore the recently published international consensus guidelines for vosoritide. These guidelines provide a roadmap for the entire treatment lifecycle, from the initial diagnosis and counseling to initiation, monitoring, and eventually, the transition off therapy.  You can review the prescribing information for vosoritide here with additional safety information, including about the risk of low blood pressure.

Joining us in-person all the way from Australia is the lead author of these guidelines, Dr. Ravi Savarirayan. Dr. Savarirayan is a global leader in skeletal dysplasia and has been a driving force in the clinical development of vosoritide from its earliest stages.

Topics Discuss:

The Journey of Vosoritide: Dr. Savarirayan shares his personal "why", from the early research phases to the clinical trials that changed the landscape of skeletal dysplasia care.

Precision Medicine at the Molecular Level: How vosoritide acts as the first precision medicine approved for achondroplasia by targeting and counteracting overactive FGFR3 signaling, along with important safety information such as a risk for low blood pressure

The International Guidelines: Why a global consensus was necessary and how it addresses gaps in real-world clinical practice.

The Treatment Lifecycle:

Counseling: Setting expectations and having the first conversation with families.

Initiation: Practical tips for daily subcutaneous injections and establishing a routine.

Safety & Monitoring: How clinicians monitor growth and manage safety considerations like hypotension across different age groups.

Discontinuation: How to navigate growth plate closure and the transition off therapy.