You must be registered for the ASBMR annual meeting to attend this presentation. 

Bone manifestations are often present in many rare disorders, impacting patient treatment and management. During this comprehensive presentation, three experts will discuss bone manifestations and therapeutic options associated with three rare skeletal conditions. Pediatric and adult Growth Hormone Deficiency (GHD) symptoms include short stature in children and reduced bone density in adults, including bone fractures in patients of all ages. Mucopolysaccharidosis (MPS) includes several types and has various bone problems that can be explained by abnormal endochondral ossification, including thoracolumbar kyphosis/scoliosis, odontoid hypoplasia, wide oar-shaped ribs, shortened long bones, coxa valga, dysplastic femoral heads, genu valgum, and bullet-shaped phalanges. Histological abnormalities have also been reported in each of the 5 zones of the growth plate. Hypochondroplasia (HCH) is a rare autosomal dominant skeletal dysplasia due to activating variants in FGFR3. It presents with disproportionate short stature with a wide range of clinical severity and is like achondroplasia, but with milder features. These characteristics include a large head, accentuated lordosis, short arms and legs, and broad, short hands and feet.

Join us as we discuss ongoing developments in the field of GHD, including long-acting growth hormone (LAGH) treatment options; increase awareness of bone pathology in MPS that impact patient bone health; and assess the importance of recognizing HCH caused by FGFTR3 mutations, allowing for improving patient outcomes through the delivery of effective management strategies and treatment options that can be immediately integrated into clinical practice.

Accreditation

EXCEL Continuing Education is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians.

EXCEL Continuing Education designates this live activity for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

You must be registered for the ASBMR annual meeting to attend this presentation. 

Educational Objectives

At the conclusion of this conference, you should be able to:

• Using sample patient cases, review the clinical presentation including bone complications associated with these three rare disorders
• Evaluate the progressive and lifelong impact that all three diseases have on bones
• Recognize the value of early and accurate diagnoses and how these may influence interventions and improved patient outcomes
• Consider the importance of optimal timing for medical treatment initiation and supportive care strategies for bone-related issues of the different types of GHD, MPS, and HCH.

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