Do you know how to differentiate between growth hormone deficiency (or GHD) and primary IGF-I deficiency (or growth hormone resistance) and the key challenges in the diagnosis and management of patients with short stature conditions?

For children presenting with short stature, an early and correct diagnosis is essential to allow children to achieve their full growth potential with appropriate treatment. Listen as expert pediatric endocrinologists delve into these rare growth disorders.

Join Prof. Philippe Backeljauw (Professor at Cincinnati Children's Hospital Medical Center and the University of Cincinnati, Cincinnati, USA) and Prof. Dr. Joachim Woelfle (Chairman of Pediatrics, Director of the Department of Pediatric and Adolescent Medicine at the Friedrich Alexander University Erlangen, Germany) as they engage in a conversation regarding key challenges in the diagnosis and management of patients with short stature conditions.

The experts discuss the incidence of patients with GHD and severe primary IGF-I deficiency and how to differentiate these conditions based on clinical presentation as well as biochemical and genetic testing. The importance of taking a good medical history and physical examination is discussed, as well as various caveats related to biochemical and genetic testing. Regional differences in the diagnosis of these patients are covered as well as key challenges in the diagnosis and management. Finally, the experts discuss the different treatment options as well as the importance of ensuring caregivers are well-informed regarding the efficacy and safety of the respective treatments.

This is the first video podcast episode in a two-part series on patients with severe short stature.

Prefer to watch as well as listen?

Watch a video of the experts in conversation and download the full transcript on the COR2ED website: https://cor2ed.com/pe-connect/programmes/rare-growth-disorders-podcast/

Or go to the video on YouTube:

https://youtu.be/OfI3lZgjew0

• Download the transcript:
  https://cor2ed.com/pe-connect/programmes/rare-growth-disorders-podcast/

• This Podcast is endorsed by the Magic Foundation. Find out more on their website: https://www.magicfoundation.org/

The medical experts in this podcast are expressing their own views and not those of COR2ED, supporters, or their institution. This podcast is supported by an Independent Medical Education Grant from Ipsen. This podcast is developed by COR2ED.com

Do you know how to differentiate between growth hormone deficiency (or GHD) and primary IGF-I deficiency (or growth hormone resistance) and the key challenges in the diagnosis and management of patients with short stature conditions?

For children presenting with short stature, an early and correct diagnosis is essential to allow children to achieve their full growth potential with appropriate treatment. Listen as expert pediatric endocrinologists delve into these rare growth disorders.

Join Prof. Philippe Backeljauw (Professor at Cincinnati Children's Hospital Medical Center and the University of Cincinnati, Cincinnati, USA) and Prof. Dr. Joachim Woelfle (Chairman of Pediatrics, Director of the Department of Pediatric and Adolescent Medicine at the Friedrich Alexander University Erlangen, Germany) as they engage in a conversation regarding key challenges in the diagnosis and management of patients with short stature conditions.

The experts discuss the incidence of patients with GHD and severe primary IGF-I deficiency and how to differentiate these conditions based on clinical presentation as well as biochemical and genetic testing. The importance of taking a good medical history and physical examination is discussed, as well as various caveats related to biochemical and genetic testing. Regional differences in the diagnosis of these patients are covered as well as key challenges in the diagnosis and management. Finally, the experts discuss the different treatment options as well as the importance of ensuring caregivers are well-informed regarding the efficacy and safety of the respective treatments.

This is the first video podcast episode in a two-part series on patients with severe short stature.

Prefer to watch as well as listen?

Watch a video of the experts in conversation and download the full transcript on the COR2ED website: https://cor2ed.com/pe-connect/programmes/rare-growth-disorders-podcast/

Or go to the video on YouTube:

https://youtu.be/OfI3lZgjew0

• Download the transcript:
  https://cor2ed.com/pe-connect/programmes/rare-growth-disorders-podcast/

• This Podcast is endorsed by the Magic Foundation. Find out more on their website: https://www.magicfoundation.org/

The medical experts in this podcast are expressing their own views and not those of COR2ED, supporters, or their institution. This podcast is supported by an Independent Medical Education Grant from Ipsen. This podcast is developed by COR2ED.com

Gene therapy and gene-modified cell therapies have a great potential for rare diseases to either help patients to cure their disease or improve their lives. Did you know that gene therapy will probably become a major treatment option for many rare diseases in the near future? Listen as expert hematologists delve into this topic and take hemophilia as a practical example.

Join Prof. Cédric Hermans (Head of the Division of Haematology, the Hemostasis and Thrombosis Unit and the Hemophilia Center of the Saint-Luc University Hospital in Brussels, Belgium) and Prof. Miguel Escobar (Professor of Medicine and Pediatrics, University of Texas Health Science Center at Houston-McGovern Medical School and University of Texas M.D. Anderson Cancer Center Medical Director, Gulf States Hemophilia & Thrombophilia Center Houston, USA) as they engage in a conversation about the potential of gene and gene-modified cell therapies in rare diseases, and how early gene therapies have been implemented as a treatment approach for rare diseases, such as leber congenital amaurosis, spinal muscular atrophy, beta-thalassemia, adrenoleukodystrophy, and hemophilia.

The experts explore hemophilia as an example of how gene therapy has become a reality within rare diseases. They discuss why we need gene therapy for rare disease, what gene therapy and gene modified cell therapy is, and how this has become a reality in hemophilia, and look to the future of gene therapy for rare diseases. Finally they reflect on the importance of a multidisciplinary approach.

- Access information on the programme, the clinical takeaways, the flashcard and the transcript here https://cor2ed.com/hemostasis-connect/programmes/gene-therapy-rare-diseases/

Find out about the experts Prof Cedric Hermans and Prof. Miguel Escobar.

The medical experts in this podcast are expressing their own views and not those of COR2ED, supporters, or their institution. This podcast is supported by an Independent Medical Education Grant from The American Society of Gene + Cell Therapy and Pfizer.