News

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

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, “…