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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…

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…

Daily treatment with AZP-3601, Amolyt Pharma’s investigational therapy for hypoparathyroidism, was well tolerated and allowed patients to discontinue standard supplement treatment, early data from a Phase 2a trial show. The treatment also rapidly normalized 24-hour urine calcium levels and improved bone health. The findings, from a first group of…

Late recovery — longer than six months — from post-surgery hypoparathyroidism is not uncommon, and having levels of parathyroid hormone (PTH) within the normal range may indicate whether patients are more likely to recover, a study found. “PTH is a useful biomarker in predicting long-term recovery of parathyroid function,”…

Severe COVID-19 could lead to the development of temporary hypoparathyroidism, a case report suggests. This report represents the third case of hypoparathyroidism secondary to severe COVID-19 infection noted in scientific literature. These rare instances, along with previous reports showing that the virus may worsen previously well-controlled hypoparathyroidism, highlight…