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…
Treatment with the injectable parathyroid hormone Natpara slows the decline in kidney function in patients with chronic hypoparathyroidism, according to recent clinical trial data. Natpara, developed by Shire, is a recombinant human parathyroid hormone (rhPTH[1-84]) approved as an add-on to the conventional treatment of chronic hypoparathyroidism with calcium and activated…
Atypical kidney lesions in adult patients may be caused by a mutation associated with a rare type of hypoparathyroidism, according to a case report. The study,“Unusual Proliferative Glomerulonephritis in a Patient Diagnosed to Have Hypoparathyroidism, Sensorineural Deafness, and Renal Dysplasia (HDR) Syndrome with…
As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…
Screening for asymptomatic hypoparathyroidism should be considered in transfusion-dependent thalassemia patients to allow for early disease detection and intervention, researchers suggest. Their study, “Under-recognized Hypoparathyroidism in Thalassemia,” was published in the Journal of Clinical Research in Pediatric Endocrinology. The occurrence of asymptomatic hypoparathyroidism, defined as low levels…
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”…
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