Routine use of Forteo (teriparatide), a synthetic parathyroid hormone fragment, appears unlikely to effectively substitute for calcium and vitamin D supplements among people with chronic hypoparathyroidism, according to the results of a Phase 3 trial. The findings were published in a study, “Chronic hypoparathyroidism and…
Entera Bio is developing a better formulation of EB612, its add-on therapy for maintaining normal blood calcium levels in people with hypoparathyroidism, and expects to launch a clinical trial next year. The biotechnology company shared its plans while announcing the publication of an earlier Phase 2a study of…
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…
Vitamin D supplementation before total thyroid removal may reduce the frequency of permanent hypoparathyroidism after surgery, a clinical study suggests. However, as the difference compared with placebo was not statistically significant, future studies with more patients, more regular follow-ups, and biochemical results are needed to better understand this interaction,…
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
Low calcium should be considered as a cause for stridor — a harsh vibrating sound made while breathing — in patients who recently had their thyroid removed. Preserving blood supply to the thyroid’s neighboring parathyroid glands after thyroid removal surgery, called a thyroidectomy, can prevent cases of postoperative hypoparathyroidism,…
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…
The presence of persistent hypoparathyroidism should prompt physicians to consider a possible diagnosis of autoimmune polyglandular syndrome type 1 (APS-1), a rare genetic disorder, even in the absence of other typical symptoms, according to a case report. The study, “Hypoparathyroidism as the single major component for decades…
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