News

Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.

Despite treatment, all patients with hypoparathyroidism experience physical and psychological symptoms that negatively impact their quality of life, a study finds. The findings were presented in a poster, “Understanding the Patient-centered Impact of Hypoparathyroidism on Functioning and Well-being,” by Ascendis Pharma during the Endocrine Society’s annual meeting,…

Contrary to other thyroid-related complications, the incidence of hypoparathyroidism in patients who undergo a thyroidectomy — a common complication associated with this type of surgery — tends to decrease between the ages of 65 and 85, a study found. The study, “Age-Related Trends of Patients Undergoing Thyroidectomy: Analysis of US Inpatient Data from…

Chronic hypoparathyroidism is linked to long-term renal and cardiovascular complications, and can have severe implications on patients’ health-related quality of life, according to data from several studies. The data, presented by Takeda Pharmaceutical during the Endocrine Society’s 2019 Annual Meeting (ENDO) in New Orleans, also included the final outcomes regarding…

Treatment with an immunotherapy regimen against skin cancer resulted in a rare case of immune-mediated hypoparathyroidism, according to a case report. This rare case was described in a study, “Inflammation-induced hypoparathyroidism triggered by combination immune checkpoint blockade for melanoma,” published in the Journal for ImmunoTherapy of Cancer.

A new study suggests that calcium supplements might not be necessary for all patients with hypoparathyroidism. This treatment approach could be especially helpful for patients dealing with calcium-related side effects. The study, “Is calcium supplementation always needed in patients with hypoparathyroidism?” was published in Clinical Endocrinology. Because hypoparathyroidism leads…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bathdissertation, she analyzed Brexit’s long-term impact…

Ascendis Pharma is planning to initiate clinical studies to evaluate the potential of its investigational therapy TransCon PTH in adult patients with hypoparathyroidism. The company has submitted an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) to start the PaTH Forward Phase 2…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…