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 $5,000 in grants, totaling…
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…
Kidney involvement, heart wall abnormalities, and left heart failure were identified as risk factors for a relapse of cardiac sarcoidosis, a long-term study involving more than 150 patients suggests. Immunosuppressant treatment with cyclophosphamide was also linked to a significantly lower risk of cardiac relapse in this patient population.
ATYR1923, a candidate therapy for pulmonary sarcoidosis, binds specifically to the neuropilin-2 (NRP2) receptor protein, which is located at the surface of immune cells responsible for inflammation and granuloma formation in lung sarcoidosis, the treatment’s developer, aTyr Pharma, has announced. These findings are described in two posters,…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
Researchers have discovered that patients with pulmonary sarcoidosis have an imbalance in two subsets of immune cells, namely B-cells and follicular T-helper cells — findings that support the potential role of autoimmune mechanisms in sarcoidosis development. The study, “Imbalance in B cell and T Follicular Helper…
The lungs of patients with sarcoidosis are burdened with a more active subset of immune T-cells, called mucosal-associated invariant T (MAIT) cells, a study reports. The activity of these MAIT cells correlates with worse disease, supporting their potential as therapeutic targets, the researchers said. The study, “…
The current scoring system used to assess the urgency of lung transplants, called LAS (lung allocation score), fails to predict the risk of death in people with sarcoidosis, a single center study suggests. The study “Mortality for sarcoidosis patients on the transplant wait list in the…
While there no therapies approved for sarcoidosis-associated pulmonary hypertension (SAPH), pulmonary hypertension-specific therapies may help ease right heart dysfunction in a select group of patients, according to results of a large cohort study. The study, “Clinical Features and Outcomes of Patients with Sarcoidosis-associated Pulmonary Hypertension,” was published in…
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