
Effective achondroplasia care depends on timely coordination across specialties, whether patients are managed at a dedicated multidisciplinary center or in the community.

Effective achondroplasia care depends on timely coordination across specialties, whether patients are managed at a dedicated multidisciplinary center or in the community.

Long-term achondroplasia care extends beyond medical treatment to supporting independence, well-being, and connection throughout the patient journey.

Response to growth-modulating therapy may evolve over several years. Structured monitoring helps clinicians assess benefit, manage expectations, and adapt treatment and orthopedic care when needed.

Investigational oral therapies are targeting the overactive FGFR3 pathway more directly, expanding the range of approaches under study for achondroplasia.

Once-weekly navepegritide provides sustained CNP exposure, with pivotal and longer-term data evaluating growth, skeletal alignment, physical function, and tolerability.

Long-term data provide insight into sustained growth, proportionality, and safety across different pediatric age groups receiving CNP analogue therapy.

Earlier intervention may offer opportunities to influence skeletal development, but treatment timing must be weighed against uncertainty, treatment burden, and the needs of individual families.

Height alone does not capture the full impact of achondroplasia. The panel discusses why health, function, proportionality, and independence are increasingly important measures of treatment success.

Beyond short stature, achondroplasia carries life-threatening risks at the skull base. The panel details why foramen magnum stenosis, hydrocephalus, and airway obstruction demand vigilant monitoring from infancy onward.

Characteristic clinical and radiographic findings can establish early suspicion for achondroplasia, while molecular and prenatal testing are helping clinicians confirm the diagnosis sooner.

Achondroplasia is driven by overactive FGFR3 signaling that disrupts normal growth plate function. The panel explains how this molecular pathway affects endochondral bone growth and provides the foundation for understanding targeted treatment.

Drs Robert Busch, Robin S. Goland, Kimberly Simmons, and Teresa Quattrin discuss strategies to facilitate screening of type 1 diabetes and improve management of disease.

Experts in endocrinology share their approach to managing type 1 diabetes along with related health conditions.

Teresa Quattrin, MD, reviews emerging clinical trial data on novel therapies to delay the progression of type 1 diabetes.

Kimberly Simmons, MD, MPH/MSPH, leads a discussion on long-term monitoring for patients with type 1 diabetes and which providers are most likely to administer and manage teplizumab treatment.

Experts in endocrinology share their approach to patient selection for teplizumab in type 1 diabetes and the impact of a 2-year delay in the onset of the disease.

Dr Kimberly Simmons reviews key findings from an initial study and extended follow-up on the safety and efficacy of teplizumab in delaying the progression of type 1 diabetes.

Robin S. Goland, MD, and Kimberly Simmons, MD, MPH/MSPH, provide insight on teplizumab, which was recently approved by the FDA, to delay the progression of type 1 diabetes, and they discuss the implications of autoantibody detection in patients.

Dr Teresa Quattrin discusses the significance of delaying the onset of type 1 diabetes.

Teresa Quattrin, MD, leads a discussion on the benefits of using medications for type 2 diabetes to manage patients who have type 1 diabetes.

Kimberly Simmons, MD, MPH/MSPH, reviews ADA guidelines recommendations for the management of type 1 diabetes.

Dr Kimberly Simmons and Dr Teresa Quattrin examine the diagnostic process and importance of screening for type 1 diabetes.

Robert Busch, MD, and Robin S. Goland, MD, review the prevalence and pathophysiology of type 1 diabetes.

February 17th 2023

February 24th 2023

February 24th 2023

February 17th 2023