Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…
More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…
Nearly 30% of scleroderma patients have nerve damage outside of the brain and spinal cord — called peripheral neuropathy — and it most frequently appears in the first decade after disease onset, according to a review study. Notably, this frequency is higher than that reported for the general…
Some patients with systemic scleroderma (SSc) appear to develop breast cancer with a good prognosis, a small study has found. The findings suggest reducing exposure to cancer therapies, namely chemotherapy and radiotherapy, which should be limited to higher-risk patients. The study, “Clinical and pathological features of breast cancer…
The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…
Nearly two-thirds of people with scleroderma undergo physical therapy — often called PT — and are generally satisfied with their care. But nearly half of all patients need more information on such treatment, according to the results of a questionnaire conducted in the Netherlands. Individual therapy, provided by…
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…
Changes from the norm in the electrical conduction of the heart — called cardiac axis deviations — are “common” and indicative of poorer outcomes in people with systemic sclerosis-related pulmonary hypertension (SSc-PH), a study reported. Because cardiac axis deviations are easily measured by electrocardiography (ECG), monitoring for such deviations…
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…
Under-the-skin treatment with tiny vesicles produced by mesenchymal stem cells significantly lessened skin abnormalities and scarring, while reducing markers of disease and inflammation, in a mouse model of scleroderma, a study showed. Notably, these beneficial effects — similar to those of treatment with the bone marrow-derived mesenchymal stem cells…
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