Patricia Inácio, PhD, science writer —

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

Removal of lymph nodes in the neck and invasion of neighboring tissues with thyroid cancer cells are risk factors for developing hypoparathyroidism following total thyroidectomy in children, a study reports. The study, “Risks of Hypoparathyroidism After Total Thyroidectomy in Children: A 21-Year Experience in a High-Volume Cancer Center,”…

A genetic screening of a large Chinese population has revealed 14 new mutations linked to the early development of hypoparathyroidism. The study, “Genetic screening in a large Chinese cohort of childhood onset hypoparathyroidism by next-generation sequencing combined with TBX1-MLPA,” was published in 
The Journal of Bone and Mineral Research.

Alizé Pharma 3, a biopharmaceutical company focused on developing therapies for rare diseases, including hypoparathyroidism, is making efforts to become a leading company focusing on rare endocrine and metabolic diseases. The company will establish operations in Europe and North America to achieve faster global regulatory approvals for leading candidate…

The U.S. Food and Drug Administration has granted orphan drug designation to TransCon PTH, an investigative therapy by Ascendis Pharma to treat hypoparathyroidism. Hypoparathyroidism is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH), resulting in low calcium and elevated phosphate levels in the blood. TransCon…