Dipak Ram (Royal Manchester Children’s Hospital UK)
LANGUAGE AND DURATION
Language: English
Duration: 30 minutes, followed by questions from the audience
PRESENTER
Presenter: Dr Dipak Ram, Consultant Paediatric Neurologist at Royal Manchester Children’s Hospital, United Kingdom
LEARNING OBJECTIVES
- Reviewing typical MLD disease trajectories and clinical characteristics
- Exploring modelling and treatment approaches available for MLD patients
- Examining the importance of early diagnosis and the early start of treatment
- Understanding the MLD patient and parent journey following the start of treatment
COURSE DESCRIPTION
Metachromatic Leukodystrophy (MLD) is a rare hereditary disease characterized by the accumulation of fats called sulfatides. Early-onset clinical manifestations are often overlooked or confused with other conditions, confusing non-trained HCPs at first contact, resulting in misdiagnosis, wrong referral routes and delays in the early start of treatment. Delays in starting treatment significantly affect the conditions’ development and the patient’s health and quality of life.
AUDIENCE
This educational activity is designed for an international audience of General Pediatricians, General Practitioners, Pediatric Neurologists, and other physicians and healthcare professionals interested in Metabolic Diseases.