News

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…

Transplant surgery that moves two or more parathyroid glands and places them elsewhere in a patient — a process called autotransplantation — can prevent postoperative hypoparathyroidism after the thyroid is totally removed in people with cancer in that gland, researchers report. Their study, “Postoperative hypoparathyroidism after total thyroidectomy for thyroid cance…

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…

Lower doses of a previously approved treatment for hypoparathyroidism are effective and safe to treat a subset of patients, according to Phase 3 clinical study. The study “Safety and Efficacy of Recombinant Human Parathyroid Hormone in Adults With Hypoparathyroidism Randomly Assigned to Receive Fixed 25-μg or 50-μg Daily Doses”…

Mirror-image versions of existing, naturally-occurring molecules called peptides, can lead to longer-lasting therapeutics, new research shows. For patients, including those with hypoparathyroidism, the discovery could translate into less frequent injections and possibly more therapies being  developed in a pill form. The study with those findings, “Method to generate highly…

Shire presented new data on its approved therapy Natpara for the treatment of patients with chronic hypoparathyroidism at ENDO 2018, the Endocrine Society’s 100th Annual Meeting and Exposition held March 17-20 in Chicago. Natpara is a recombinant human parathyroid hormone (rhPTH) that’s designed to replace the natural hormone, called PTH,…

TransCon PTH, an investigational therapy for hypoparathyroidism (HP) being developed by Ascendis Pharma, showed positive results in healthy volunteers, according to initial data from a Phase 1 clinical trial. The company presented the trial data recently at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco, California. “We look forward to…

Shire plans a Phase 4 clinical trial of its parathyroid hormone therapy Natpara as an add-on to standard hypothyroidism treatment. Hypoparathyroidism is caused by abnormal low levels of parathyroid hormone (PTH), which regulates levels of calcium in blood and bones. Low levels of the hormone lead to a  mineral imbalance that…

Treatment of patients with pseudohypoparathyroidism type 1B should involve lowering parathyroid hormone levels to avoid adverse effects on bone, according to a recent study. Pseudohypoparathyroidism (PHP) is a heterogeneous group of rare endocrine disorders characterized by normal renal function and resistance to the action of the parathyroid hormone — a hormone…