Opening remarks

A Critical Review of Somapacitan Data in GHD

First Clinical Experience in Managing Children with GHD Treated with Somapacitan

Q&A

Closing remarks

Welcome and introductions

Optimising multidisciplinary team management in rare bone diseases

A collaborative approach to managing patients with rare bone diseases: Interactive case studies discussing XLH, OI and others

Summary, Final Questions and Close

Welcome and introduction

Natural course and genetic etiologies

Q&A

Diagnosis of delayed puberty versus congenital hypogonadotropic hypogonadism

Q&A

Old and emerging therapies

Q&A session

Panel discussion and meeting close

Welcome and introductions

Genetic impairments in the MC4R pathway and their role in hyperphagia and early-onset, severe obesity

Overview of MC4R pathway diseases, including Bardet-Biedl syndrome (BBS)

The role of precision medicine in the management of BBS and other MC4R pathway diseases

Group discussion: Diagnosis and genetic testing

Summary and close

Welcome, introduction, and objectives

Patient and caregiver perspectives in pGHD

The evidence to date: Long term data from the Somatrogon Clinical Trial Program

Somatrogon in practice: Patient Case Studies

Panel Discussion

Leveraging knowledge from diabetes ecosystem for innovation in digital health technologies

User centered approach in assessment of connected autoinjectors for patients requiring GH therapy

Current perspectives on evidence based therapeutic interventions from real world data in growth hormone therapy

Panel Discussion

Introduction and objectives

Growing up with vosoritide▼: Long-term efficacy in children of different ages.

From trials to treatment: Real-world experience with vosoritide

Q&A and closing remarks

▼ This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. Healthcare professionals should report adverse events in accordance with their local requirements. Adverse events should also be reported to BioMarin on + 1 415 506 6179 or [email protected].

Introduction and the complex pathophysiology of leptin deficiency

The early onset burden of lipodystrophy: lessons from the paediatric population

Treatment approaches to the child with lipodystrophy

Patient education initiatives in lipodystrophy: learnings from the LIPEA program

Conclusions and Q&A