Hereditary Angioedema Hereditary angioedema (HAE) is a rare disease which causes swelling episodes throughout the body, including the airways, gastrointestinal system, and extremities. Most commonly, HAE is caused by a…
According to a story from Angioedema News, a team of researchers and scientists have successfully developed a tool that is able to measure disease activity in hereditary angioedema that is…
According to a story from newswiretoday.com, the biopharmaceutical group Ipsen has recently announced its decision to halt its dosing of patients in its phase 3 clinical trial and its phase…
According to a story from Malaysian Tribune, the pharmaceutical company Khondrion has just announced that the first patients have been dosed in its phase IIb clinical trial. This trial is…
Ipsen is a pharmaceutical company based out of Paris. Sadly, they have just announced that they have pressed pause on not one but two studies for fibrodysplasia ossificans progressiva (FOP). FOP…
Proteostasis Therapeutics has just announced results from their ex-vivo study of PTI CFTR modulators using organoids from patients living with cystic fibrosis (CF). These organoids are genetically identical, and have…
Khondrion has just announced that the very first patient has been dosed in their Phase 2b study for mitochondrial diseases called KHENERGYZE. This patient population has a high unmet need…
.png)
Researchers at the Medical Center of Hackensack University conducted a study of three drugs now proven to be safe and effective for the treatment of severe seizures. According to…
The Benefits of Genomics The use of genomics in health systems is becoming more of a standard practice. Health systems have been partnering with genomics databases to collect patient data.…
FightMND is an Australian nonprofit organization dedicated to improving the lives of motor neurone disease patients through funding research for cures. They've successfully provided millions toward research initiatives and clinical…
International Alagille Awareness Day The very first International Alagille Awareness day was held on January 24th, 2020. What is Alagille syndrome? It is a rare disease which effects the heart,…
According to a story from consultant360.com, a recent study was conducted in order to determine if racial ancestry had any impacts on outcomes in the rare disease pulmonary arterial hypertension…
Seelos Therapeutics has won a meeting with the European Medicines Agency (EMA) to advance its therapy, trehalose, for the treatment of neuro-degenerative diseases, specifically Sanfilippo syndrome. At this meeting, which…
The Platform Notable's automated technology platform was created to help predict which patients would respond better to which therapies. As no patient is the same, this study is paramount. Notable…
According to a story from Federal Telemedicine News, there are around 7,000 diseases known to science that can be considered a rare disease. When we consider these diseases in the…
.jpg)
According to a recent article in MedPage Today’s 2019 year-end review, the number of life-threatening lung diseases in systemic juvenile idiopathic arthritis (sJIA) patients has been increasing. sJIA is…
According to a story from Medical Xpress, a recent study has concluded that characteristics of a patient's lung microbiome can help predict treatment outcomes, particular in patients with severe lung…
More than 20 years ago researchers at Harvard University, led by professor Oliver Pourquié, discovered how the vertebra in chickens first form. Essentially, a 'tick' initiates the formation of a…
Epithelioid sarcoma is a rare form (accounts for <1%) of soft tissue sarcoma. Until now, there have been no treatments specifically for this patient population. But the FDA has just…
According to a story from Medical Xpress, a recent study that draws on 30 years of maple syrup urine disease patient data has concluded that while significant improvements have been…
According to a story from Medical Xpress, a team of researchers from the medical school at the University of Massachusetts has revealed new information about the nature of neurodegenerative symptoms…
SELLAS has just announced that patient screening has begun for a Phase 3 trial for acute myeloid leukemia (AML). The trial is examining the effects of GPS for patients who…
It has been evident to scientists for years that Alzheimer’s disease, which affects about five million people in the U.S., is caused by an accumulation of two proteins within…
According to a story from Newswise, a team of scientists and researchers associated with Université de Montréal and CHU Sainte-Justine have recently completed a study that maps a cellular model…
.jpg)
According to a recent article in the UK Newcastle Chronicle, when doctors gave Leanne Buckley an opportunity to terminate both her pregnancies, Leanne and her husband Michael were emphatic…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
