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An 86-year-old woman with hypoparathyroidism and progressive cognitive deficits was found to have calcium deposits in both hemispheres of her brain, according to a case report. The study, “Hypoparathyroidism Associated with Vascular Centrum Semiovale Calcifications,” was reported in the journal Annals of Neurology. Hypoparathyroidism is typically associated…

TransCon PTH normalized calcium levels in the blood and urine of adults with hypoparathyroidism and eliminated the need for vitamin D supplements and high doses of calcium, according to more than 1.5 years of trial data. This data — 84 weeks’ worth in all — came from the…

Replacement therapy with parathyroid hormone (PTH) — the hormone found at low levels in patients with hypoparathyroidism — requires the presence of another hormone called fibroblast growth factor 23 (FGF23) to help the kidneys expel excess phosphate, a small study has found. The study, “PTH and FGF23 exert…

Over 230 national organizations signed a letter urging all 50 U.S. state governors to “maintain and expand” flexibility with licensure requirements for the duration of the COVID-19 pandemic to ease access to care. During the pandemic, governors used emergency authority to waive certain state licensure requirements, giving healthcare providers…

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than for those without rare diseases. This study provides evidence of the potential effect rare diseases may have on public health…

The American Thyroid Association (ATA) diagnostic criteria for postoperative hypoparathyroidism are useful to doctors in grouping patients based on symptoms, which could help in managing the disease, a study reported. “However,” the researchers wrote, differentiating patients in this way “may underestimate the actual incidence,” or the number of…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

AZP-3601, an investigational treatment for chronic hypoparathyroidism, was able to safely increase blood calcium levels in healthy volunteers in a Phase 1 clinical trial, top-line data show. “We are pleased with these clinical trial results for AZP-3601 … as they support a potential therapeutic profile that we believe can address…

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…