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…
A routine procedure called PGA — for parathyroid gland autotransplantation — often done following surgery to remove the thyroid gland, is associated with a higher risk of immediate hypoparathyroidism, a study shows. However, PGA did not raise the risk of permanent hypoparathyroidism, or that lasting more than six months.
Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…
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…
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…
In a woman whose severe hypoparathyroidism did not respond to standard treatment, constant infusion of a lab-engineered form of the parathyroid hormone (PTH) eliminated symptoms and improved her quality of life, a case study from Portugal reports. This medication, called Forsteo (teriparatide) and marketed in the U.S. as…
Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…
The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
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