Patricia Inácio, PhD,  science writer—

Daily treatment with AZP-3601, Amolyt Pharma’s experimental therapy for hypoparathyroidism, safely and effectively normalizes calcium levels in people with the disease, according to data from the second group of patients in a proof-of-concept Phase 2a clinical trial. Most participants stopped calcium and vitamin D supplements, which is the…

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…

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…

Six months of treatment with Forteo (teriparatide) — a lab-made parathyroid hormone fragment approved for osteoporosis — safely led to calcium and phosphate control in a 4-month old girl with severe hypoparathyroidism, a case report found. These findings highlight the potential of this off-label medication to treat infants with…

Impaired activity of the adrenal glands, a condition called Addison’s disease, may be the only typical manifestation of autoimmune polyglandular syndrome type 1 (APS1) to have occurred decades after diagnosis, a case report suggests. APS1, a rare inherited autoimmune disorder, is typically characterized by at least two of three manifestations:…

Children with thyroid cancer or disease who undergo surgery to remove that gland can commonly have their parathyroid glands accidentally removed during the thyroidectomy, a U.S. study reports. While such surgeries are rare in children, this finding calls for meticulous attention from surgeons performing a thyroidectomy — especially if examining…

Amolyt Pharma has raised $80 million in financing that will support the clinical development of AZP-3601, its experimental therapy for hypoparathyroidism. Hypoparathyroidism is caused by abnormally low levels of the parathyroid hormone (PTH), leading to low vitamin D and calcium levels, as well as high phosphorus levels…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…