Ascendis Pharma anticipates it will announce top-line data from a new Phase 3 clinical trial of TransCon PTH, the company’s investigational hormone replacement therapy for hypoparathyroidism, before the end of the year, according to a recent company update. The study, called PaTHway (NCT04701203), seeks to assess the…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AZP-3601, an investigational therapy for hypoparathyroidism by Amolyt Pharma. The designation is given to treatment candidates with the potential to be safe and effective in rare diseases, defined in the U.S as those affecting fewer than…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…
People with hypoparathyroidism in Argentina, particularly those with kidney problems and high levels of phosphate in the blood, frequently experience inadequate disease control, a study found. The study is the first to analyze hypoparathyroidism in Latin America, researchers said. Titled “Hipopara-Red, Real Life Experience in 322…
Long-term treatment of hypoparathyroidism with Forteo (teriparatide), a lab-made version of the parathyroid hormone, was safe and reduced the need for supplemental therapies in what researchers describe as the first case of the condition in a child with Jacobsen syndrome (JS). The report,…
Immune checkpoint inhibitors (ICIs), a type of immune therapy used to treat some cancers, may lead to the development of immune-related hypoparathyroidism, according to a case report. Although rare, this case adds to three other similar cases and to an increasing number of reports describing immune-related hormone diseases…
To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…
People with chronic hypoparathyroidism not controlled by conventional therapy may experience a substantial burden of illness and impaired quality of life, mostly due to persistent symptoms and multiple co-existing conditions. That finding comes from a survey-based study of attending physicians, “…
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…
The specific surgical procedures used when patients undergo thyroid surgery — known as thyroidectomy — dictate the risks they have of developing hypoparathyroidism and other complications, a study found. Because this type of surgery is associated with a high risk of complications and requires specialized care, it should…
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