Kohl's has brought a new clothing line whose target audience is rare disease patients. The new clothing includes adaptive options to allow easy access to medical ports and easy dressing.…
BPD Bronchopulmonary dysplasia (BPD) is a rare condition that primarily affects the lungs of premature babies (though it can affect full term newborns as well). Most children with BPD are born…
Immunoglobulin A nephropathy (IgAN) is a glomerular disease which can lead to kidney failure. Between 70 and 100 percent of all IgAN patients also are diagnosed with microscopic hematuria, but…
Research During COVID Many medical research projects have faced hurdles since the pandemic has started. Regulators have changed development timelines, and many have had to delay their clinical trials due…
The Partnership A new partnership has just been announced between Mendelian, a digital health company for rare disease, and Modality NHS partnership, a rare disease diagnosis program. Together, these organizations…
Eurordis The European Organization for Rare Diseases (Eurordis) is a nonprofit organization which organizes over 930 rare disease groups into one alliance. Groups have joined the network from 73 different countries.…
Laura Bonnell’s two children have cystic fibrosis (CF). This condition affects the respiratory system and can severely impact patient’s immune system. She explains how she didn’t own a single N95…
Spinal Muscular Atrophy Spinal muscular atrophy (SMA) is a rare disease that causes progressive muscle loss and resultant muscle weakness. It is caused by a lack of motor neurons. It…
Throughout the COVID-19 pandemic, researchers have been dedicated to understanding which individuals are most at risk for severe disease. They've found that those who are immunocompromised and have certain cancers…
Blueprint Medicines Corporation has just announced that they have submitted a supplemental New Drug Application (NDA) to the FDA. This application is for a therapy called AYVAKIT, which was developed…
Researchers have uncovered a new way to treat cancer that could lessen the side effects that patients often experience from chemotherapy. At the same time, it improves the curative power…
No deal Brexit means that the UK will be excluded from the 24 ERNs (European Reference Networks). Without the sharing of information, data, and research across countries, rare disease research…
Pulmonary fibrosis is a condition that is caused by scarring of the lungs which can lead to shortness of breath and other respiratory issues. Some cases of COVID-19 have lead to…
Many rare disease patients understand the financial burdens of living with a rare disease. As the therapies are only supporting a small number of patients, the price tag is often…
Complex Regional Pain Syndrome Complex regional pain syndrome (CRPS) is a rare condition that causes prolonged pain after a minor injury. This pain is severe and can be debilitating. One…
As the pandemic has raged on, we have learned more and more about what side effects can occur from severe COVID-19 infection. Some of these are common in patients with…
Researchers have uncovered that there is a link between Alzheimer's disease and three eye conditions- glaucoma, diabetic retinopathy, and macular degeneration (age-related). This could mean that there may be another…
There is a large stigma against those with disabilities. Many misunderstand the wide diversity in disability and the success that those who are disabled can have in their lives. This…
Transitions to adult care for patients who have a chronic respiratory disease, like cystic fibrosis, or a type of neuromuscular disease that influences the respiratory system is something that the medical…
The American College of Rheumatology presented new findings on November 5, 2020 at their virtual conference. They found that in patients with ANCA-associated vasculitis (AAV), hypothyroidism is associated with positive…
The Orphan Drug Act originated in 1983 as an incentive for pharmaceutical companies who worked on developing treatments for rare disease. Since rare diseases are well, rare, choosing to work…
We all know that diagnosing cancer earlier means better outcomes for patients. It’s also essential to monitor the spread of cancer in already diagnosed patients. If we can watch as…
Michael George and his team from the University of Pennsylvania have recently uncovered new insights regarding autoimmune patient’s comfortability with care during COVID-19. They've been working since the pandemic began…
Dilated cardiomyopathy is a heart condition caused by a mutation in the RBM20 gene. This heart condition is unique because it can affect young adults. These individuals are at an…
PMDD Premenstrual dysphoric disorder (PMDD) is a psychological condition which cyclically occurs with the menstruation cycle. Unfortunately, because the symptoms of this condition can mirror many mental health diagnoses, it…
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