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Ascendis Pharma has submitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of its treatment candidate TransCon PTH for adults with hypoparathyroidism, the company announced. “Our data support that TransCon PTH, if approved, could become the first-in-class therapy to address the underlying cause of…

Patients with long-lasting hypoparathyroidism in Sweden are over three times more likely than healthy individuals to develop kidney complications such as chronic kidney disease and kidney stones, and this may translate into more frequent hospital admissions, a study found. The study, “Kidney Complications and Hospitalization in Patients with…

Patients with chronic hypoparathyroidism following surgery for thyroid cancer do not have overall significant cognitive impairments, but those with lower blood calcium levels showed some disruptions in cognitive function, according to a study. Specifically, patients with lower calcium levels exhibited impairments in visuo-spatial attention, semantic memory (memory of general…

Adults with hypoparathyroidism given long-term treatment with Natpara are less likely to experience heart-related health problems, according to an analysis of data from three clinical trials. Natpara “may confer cardiovascular benefits to patients with chronic hypoparathyroidism, and further research is warranted to understand the relationship better in a real-world setting,” the researchers…

MBX 2109, an experimental hormone replacement therapy that MBX Biosciences is developing for hypoparathyroidism, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The FDA awards this designation to therapies intended to treat rare diseases, defined as those that affect less than 200,000 people…

Replacement therapy with either Forteo or Natpara, two versions of the parathyroid hormone (PTH), appears to be safe and effective in controlling calcium and phosphorus pools in the body, and this translates to better quality of life for patients with hypoparathyroidism, a meta-analysis study has found. The study, “…

Calcium citrate was as effective as calcium carbonate at sustaining normal calcium levels in people with hypoparathyroidism, but was associated with fewer gastrointestinal side effects and a potentially lower risk of nephrolithiasis, or kidney stones, according to a study. “These results are likely to have clinical implications in [hypoparathyroidism],…

Note: This story was updated July 13, 2022, to correct the name of Rare-X’s CEO Charlene Son Rigby. Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world.

The HypoPARAthyroidism Association has launched the first-of-its-kind Patient-Powered Data Platform, a repository to help understand the disease journey and develop better treatments for people with hypoparathyroidism. Interested participants — patients, caregivers, or those who have lost a loved one to the rare disease — can enter patient…

Children successfully treated for a cancer who undergo thyroid surgery for a secondary thyroid disease have a similar risk of surgical complications, such as permanent hypoparathyroidism, as children without a previous cancer, a single-center study in the U.S. shows. These findings add to an ongoing discussion of whether cancer…