News

ATA Criteria Useful for Grouping Patients by Hypocalcemia Symptoms

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, Diversity Coalition Team Up to Advance Health Equity

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…

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

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…

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

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…

Amolyt Raises $80M to Develop AZP-3601 for Hypoparathyroidism

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…

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…