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As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…
A single dose of AZP-3601 — Amolyt Pharma’s experimental therapy for hypoparathyroidism — resulted in a significant, sustained increase in blood calcium levels for at least 24 hours in healthy volunteers, according to interim data from a Phase 1 trial. These early findings support the therapeutic potential of…
COVID-19 may lead to the deterioration of some medical conditions, such as hypoparathyroidism, that had been well controlled before the infection, a case report suggests. The study, “Decompensated primary hypoparathyroidism in a patient with COVID-19,” was published in the journal Annales d’Endocrinologie. Hypoparathyroidism is…
Aeterna Zentaris has entered into a licensing agreement with the University of Sheffield to develop a potential therapy for adults with primary hypoparathyroidism, according to a recent press release. The treatment candidate is based on parathyroid hormone (PTH) fusion polypeptides — chains of amino acids, the building blocks…
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…
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…
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