For Health Care Professionals

ONLINE COURSE:

Challenges in Diagnosis and Disease Management of MPS and alpha-mannosidosis

The free-to-attend online course has been designed to help specialists and general paediatricians to reduce delay in the referral and diagnosis of patients with suspected MPS and alpha-mannosidosis, as well as improving the quality of patient’s lives who have already been diagnosed.

On-time diagnosis is crucial for access to treatment and clinical trials, disease management and to access the proper care services at the earliest of the disease possible. This subsequently leads to a better quality of life for the patients, and as well for their families and caregivers.

The course is free to attend, and a certificate of attendance will be provided by the Excellence in Pediatrics Institute for those who attend each module of the course. As soon as you register for the selected module you will also be registered for all the modules of the course. Further information on all upcoming modules as part of course, below:

An MPS Europe project in partnership with MetabERN and the Excellence in Pediatrics

Module 1:Introducing MPS and Alpha-mannosidosis: Challenges in Diagnosis and Disease Management

Christina Lampe is Director of the Centre for Rare Diseases of the Department of Child Neurology, Epileptology and Social Medicine, Center for Paediatric and Adolescent Medicine, University Hospital of Gießen, Germany.

Organised: Wed, Dec 18, 2019
Time: 1:00 pm – 2:00 pm CET (Central European Time)
Presenter: CHRISTINA LAMPE

You can still attend the session. Click Here to Register and watch the recorded video. It’s free
  • Duration: 60 min split into two parts (20 min presentation and 10 min of Q&A followed by 20 min presentation and 10 min of Q&A)
  • Language: English
  • Audience: Pediatricians, General Practitioners, Specialists.
Module Learning Objectives

PART 1: Introduction and Overview – What is MPS & Alpha-mannosidosis? (1) How to diagnose MPS/mannosidosis early (early, signs and symptoms) (2) Exploring the latest diagnostic options available to you (3) Reviewing the pathophysiology of MPS /mannosidosis

PART 2: Red flags, Signs and symptom, the basis of diagnostics and when to refer if you suspect MPS or Alpha-mannosidosis (4) Examining the multi-systemic features of the diseases (5) Reviewing how to best follow up with patients – what are the immediate next steps? (6) Understanding the latest treatment options available to the patient.

 

Important Note: The information provided on this session is intended and designed for use by healthcare professionals with an interest in metabolic diseases, MPS and alpha-mannosidosis. All session viewers are required to register for the selected webinar before accessing the content. Content and views shared are solely those of the speaker.

The free-to-attend online course has been designed to help specialists and general paediatricians to reduce delay in the referral and diagnosis of patients with suspected MPS and alpha-mannosidosis, as well as improving the quality of patient’s lives who have already been diagnosed. On-time diagnosis is crucial for access to treatment and clinical trials, disease management and to access the proper care services at the earliest of the disease possible.

The course is an MPS Europe project in partnership with MetabERN and the Excellence in Pediatrics with the support of Biomarin, Chiesi, Orchard Therapeutics, Regenxbio, Sanofi Genzyme, Takeda, and Ultragenyx. All modules of the course are independently organised by EIP in terms of content, topics and speakers appointed. 

Module 2: MPS and Alpha-mannosidosis:
Challenges in Disease Management – Otorhinolaryngology and Anaesthesiology

Mehmet Umut Akyol is Professor of Otorhinolaryngology, Hacettepe University, Turkey

Matthias K. Schaefer is working in the Clinic for Anesthesia, Intensive Care, Emergency Medicine and Pain Therapy, Acura-Kliniken, Albstadt, Germany. Honorary Clinical Lecturer at the University Medical School of Mainz, Mainz, Germany and the University of Jena, Germany.

Organised: Wed, Jan 29, 2020
Time: 1:00 pm – 2:00 pm CET (Central European Time)
Presenter 1: MEHMET UMUT AKYOL
Presenter 2: MATTHIAS SCHAFER

You can still attend the session. Click Here to Register and attend the recorded video. It’s free
  • Duration: 30 min(split into two parts followed by Q&A)
  • Language: English
  • Audience: Pediatricians, General Practitioners, Otorhinolaryngologists, and Anesthesiologists
Module Learning Objectives

PART 1 – OTORHINOLARYNGOLOGY: (1) Gain awareness about the main otorhinolaryngological presenting signs of mucopolysaccharidoses and Alpha-mannosidosis and their time of show-up. (2) Recognize the challenges and dangers of surgical treatment of patients with mucopolysaccharidosis and Alpha-mannosidosis. (3) An overview of the other Otorhinolaryngological presenting symptoms of MPS and Alpha-mannosidosis and recommendations about the timing of treatment, treatment/consultation options.

PART 2 – ANAESTHESIOLOGY: (1) Gain awareness about the adequate perioperative workup of severe phenotype MPS-patients. (2) Recognize the challenges and dangers of anaesthesia management of patients with MPS (3) An overview of main MPS symptoms influencing anaesthesia and postoperative care and recommendations for management.

Important Note: The information provided on this session is intended and designed for use by healthcare professionals with an interest in metabolic diseases, MPS and alpha-mannosidosis. All session viewers are required to register for the selected webinar before accessing the content. Content and views shared are solely those of the speaker.

The free-to-attend online course has been designed to help specialists and general paediatricians to reduce delay in the referral and diagnosis of patients with suspected MPS and alpha-mannosidosis, as well as improving the quality of patient’s lives who have already been diagnosed. On-time diagnosis is crucial for access to treatment and clinical trials, disease management and to access the proper care services at the earliest of the disease possible.

The course is an MPS Europe project in partnership with MetabERN and the Excellence in Pediatrics with the support of Biomarin, Chiesi, Orchard Therapeutics, Regenxbio, Sanofi Genzyme, Takeda, and Ultragenyx. All modules of the course are independently organised by EIP in terms of content, topics and speakers appointed. 

Module 3: MPS and Alpha–mannosidosis: Challenges in Disease Management – Orthopaedics

Andrea Borgo is Orthopaedic Surgeon and Assistant Professor at the Orthopaedic Clinic, University General Hospital of Padova, Italy

Organised: Wed, Feb 5, 2020 
Time: 1:00 pm – 2:00 pm CET (Central European Time)
Presenter: ANDREA BORGO

You can still attend the session. Click Here to Register and watch the recorded video. It’s free
  • Duration:30 min followed by Q&A
  • Language: English
  • Audience: Pediatricians, General Practitioners, Orthopedists
Module Learning Objectives

(1) Know what are the musculoskeletal manifestations of mucopolysaccharidoses and Alpha-mannosidosis
(2) Recognize some precocious musculoskeletal signs suggestive for a metabolic disease in order to send the patients to the right diagnostic pathway
(3) Understand the orthopaedic surgical options (and their timing) in a child affected by mucopolysaccharidoses.

Important Note: The information provided on this session is intended and designed for use by healthcare professionals with an interest in metabolic diseases, MPS and alpha-mannosidosis. All session viewers are required to register for the selected webinar before accessing the content. Content and views shared are solely those of the speaker.

The free-to-attend online course has been designed to help specialists and general paediatricians to reduce delay in the referral and diagnosis of patients with suspected MPS and alpha-mannosidosis, as well as improving the quality of patient’s lives who have already been diagnosed. On-time diagnosis is crucial for access to treatment and clinical trials, disease management and to access the proper care services at the earliest of the disease possible.

The course is an MPS Europe project in partnership with MetabERN and the Excellence in Pediatrics with the support of Biomarin, Chiesi, Orchard Therapeutics, Regenxbio, Sanofi Genzyme, Takeda, and Ultragenyx. All modules of the course are independently organised by EIP in terms of content, topics and speakers appointed. 

Module 4: MPS and Alpha–mannosidosis: Challenges in Disease Management – Neurology

Maurizio Scarpa is Director of the Regional Coordinating Center for Rare Diseases Udine, University Hospital Udine, Italy

Organised: Wed, Feb 12, 2020 
Time: 1:00 pm – 2:00 pm CET (Central European Time)
Presenter: MAURIZIO SCARPA

You can still attend the session. Click Here to Register and watch the recorded video. It’s free
  • Duration: 45 min (30-min webinar and 15-min update and panel discussion).
  • Language: English
  • Audience: Pediatricians, General Practitioners, Neurologists
Module Learning Objectives

(1) Understand the pathogenesis of CNS involvement in MPS and Alpha-mannosidosis
(2) Learn clinical neurological features on MPS and Alpha-mannosidosis
(3) Exploring the principle of neurological therapy in MPS and Alpha-mannosidosis

Important Note: The information provided on this session is intended and designed for use by healthcare professionals with an interest in metabolic diseases, MPS and alpha-mannosidosis. All session viewers are required to register for the selected webinar before accessing the content. Content and views shared are solely those of the speaker.

The free-to-attend online course has been designed to help specialists and general paediatricians to reduce delay in the referral and diagnosis of patients with suspected MPS and alpha-mannosidosis, as well as improving the quality of patient’s lives who have already been diagnosed. On-time diagnosis is crucial for access to treatment and clinical trials, disease management and to access the proper care services at the earliest of the disease possible.

The course is an MPS Europe project in partnership with MetabERN and the Excellence in Pediatrics with the support of Biomarin, Chiesi, Orchard Therapeutics, Regenxbio, Sanofi Genzyme, Takeda, and Ultragenyx. All modules of the course are independently organised by EIP in terms of content, topics and speakers appointed. 

Module 5: MPS and Alpha–mannosidosis: Challenges in Disease Management – Cardiology

Dr Elizabeth Braunlin is a Professor of Pediatrics in the Division of Pediatric Cardiology and Director of the Pediatric Cardiology Fellowship Program. She received her M.D. from the University of Minnesota. Dr Braunlin completed her residency in Pediatrics at The Children’s Hospital Medical Center in Boston and a fellowship in Pediatric Cardiology at the University of Minnesota. Dr Braunlin completed her PhD in Physiology at the University of Minnesota. She joined the faculty at the University of Minnesota in 1983. She is board certified in Pediatrics and Pediatric Cardiology.

Dr Braunlin’s clinical interests are prevention and treatment of post-transplant lymphoproliferative disease (PTLD) after cardiac transplantation; the long term cardiac follow-up of individuals with mucopolysaccharidoses after marrow transplantation or enzyme replacement therapy; and prevention and management of cardiac failure in individuals with various forms of muscular dystrophy. Her basic research interest involves investigation by cardiac ultrasound and histopathology of murine models of mucopolysaccharidoses both before and after novel therapies aimed at correction. 

 
 
Date: Wed, Feb 19, 2020  Time: 1:00 pm – 2:00 pm CET (Central European Time) Presenter: ELIZABETH BRAUNLIN, Professor, Department of Pediatrics, Division of Pediatric Cardiology, Team Member of Mucopolysaccharidosis (MPS) Center, University of Minnesota, USA
  • Duration: 30 min (20 min presentation followed by 10 min Q&A)
  • Language: English
  • Audience: Pediatricians, General Practitioners, Cardiologists

Module Learning Objectives: (1) Develop an understanding of the cardiac manifestations of MPS (2) Understand the methods of assessing the cardiac manifestations of MPS (3) Understand the effects of ERT and HCT on the cardiac manifestations of MPS.

Important Note: The information provided on this session is intended and designed for use by healthcare professionals with an interest in metabolic diseases, MPS and alpha-mannosidosis. All session viewers are required to register for the selected webinar before accessing the content. Content and views shared are solely those of the speaker.

The free-to-attend online course has been designed to help specialists and general paediatricians to reduce delay in the referral and diagnosis of patients with suspected MPS and alpha-mannosidosis, as well as improving the quality of patient’s lives who have already been diagnosed. On-time diagnosis is crucial for access to treatment and clinical trials, disease management and to access the proper care services at the earliest of the disease possible.

The course is an MPS Europe project in partnership with MetabERN and the Excellence in Pediatrics with the support of Biomarin, Chiesi, Orchard Therapeutics, Regenxbio, Sanofi Genzyme, Takeda, and Ultragenyx. All modules of the course are independently organised by EIP in terms of content, topics and speakers appointed.