News

Ascendis Pharma’s investigational therapy TransCon PTH safely restored calcium and phosphate balance and eliminated the need for current standard care in adults with hypoparathyroidism, top-line data from a Phase 2 clinical trial show. “With TransCon PTH, our goal has always been to improve the lives of patients…

People needing to take vitamin D and calcium supplements due to very low levels soon after thyroid cancer surgery are likely to experience a decline in their quality of life (QOL) over at least the following year, a study suggests. As such, assessing calcium and vitamin D levels, as well…

Undetectable parathyroid hormone (PTH) levels in the blood one day after total thyroidectomy and removal of lymph nodes in the neck is a significant risk factor to develop permanent hypoparathyroidism, researchers suggest. The study, “Risk factors of permanent hypoparathyroidism after total thyroidectomy and central neck dissection for…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

Long-term treatment with Takeda’s parathyroid hormone therapy Natpara normalizes bone remodeling and bone density in people with hypoparathyrodism, according to a four-year study. This is the longest study of Natpara’s effects on bone structure in this patient population, as assessed with a non-invasive method called high…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

A novel mutation in the TBX1 gene caused hypoparathyroidism and deafness in a man and his three children, a case study reports. The study, “A Novel TBX1 Variant Causing Hypoparathyroidism and Deafness,” was published in the Journal of the Endocrine Society. Hypoparathyroidism is caused by low levels of…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

Patients with DiGeorge syndrome tend to experience hypoparathyroidism symptoms earlier — and ones that are milder — compared to those with idiopathic hypoparathyroidism, a study has found. The study, “Clinical and Genetic Findings in a Chinese Cohort of Patients With DiGeorge Syndrome-Related Hypoparathyroidism,” was published in…

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…