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Endocrinology

We work in three rare endocrinology diseases

We work in three rare endocrinology diseases

Overview

TransCon hGH (lonapegsomatropin)

Growth Hormone Deficiency (GHD)

GHD is a rare growth disorder that affects not only height but overall health and development. Children with GHD are characterized primarily by growth failure, as well as metabolic abnormalities, psychosocial challenges and poor quality of life.

GHD in adults is associated with abnormal metabolism and body composition, as well as psychological difficulties. If untreated in patients with these symptoms, GHD in adults may lead to decreased bone mineral density, increased risk of cardiovascular mortality, and poor quality of life.

Adult Hypoparathyroidism

Hypoparathyroidism is defined as a decrease or lack of parathyroid hormone, leading to low levels of calcium in the blood and an increase in serum phosphates1.

Calcium and/or active vitamin D can help relieve symptoms, but these treatments can have undesirable side effects including an increased risk of kidney stones and chronic kidney disease1.

Achondroplasia

Achondroplasia is a skeletal disease and the most common form of dwarfism. Those living with the condition may experience severe skeletal complications and comorbidities.

Abnormal skeletal development can lead to sleep apnea, chronic back and leg pain from lower spine impingement and sudden infant death from cervical compression. Chronic ear infections due to abnormal eustachian tubes can lead to hearing loss and speech delay.

  1. Mannstadt, M., Bilezikian, J.P., Thakker, R.V., Hannan, F.M., Clarke, B.L., Rejnmark, L., Mitchell, D.M., Vokes, T.J., Winer, K.K. and Shoback, D.M. (2017). Hypoparathyroidism. Nature Reviews Disease Primers, 3(1). doi:https://doi.org/10.1038/nrdp.2017.55.

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