The State of PTO Of all of the industrialized countries in the world, the United States is the only one that in the year 2020 still doesn't guarantee that workers…
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…
As originally reported in NewsRoom, the US's political volatility over the past years has thrown many policies and legislation into debate. While switching the party in both the executive and…
When Lily Wythe, a 14-year-old from Eastwood in England, was diagnosed with diffuse pontine glioma (also known as DIPG), her friends and family knew they had to do something to…
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According to a recent article in the MalayMail, it occurred to Abdullah’s doctor that the 67-year-old would be an inspiration for others who have kidney failure. His nephrologist, Dr.…
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,…
As originally reported in Euronews, there is limited supply of expensive medicines for rare diseases, so it can be difficult to prioritize which patients will have their lives saved when…
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…
In a post from MD Magazine, a video from the HCPLive Network depicts a conversation between a number of sickle cell anemia experts about different clinical presentations and complications associated…
India first drafted a national rare disease policy back in 2017. Unfortunately, it was never enacted due to budgeting and implementation issues. This draft included suggestions such as a corpus…
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…
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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…
A correct diagnosis is often difficult to obtain, especially for a rare disease. Symptoms can be attributed to more common conditions, or they can be written off entirely. Rylee…
30 Things About My Rare Invisible Illness You May Not Know By Debra Richardson 1. The illness I live with is: stiff person syndrome & insulin-dependent diabetes. 2. I was…
On April 28th, 2020, Acromegaly Community, a nonprofit organization that is dedicated to supporting acromegaly patients, will be hosting a patient focused drug development meeting (PFDD). These meetings are an…
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…
By Rachel Whetstone from In The Cloud Copy The Orphan Drug Act of 1983 was created to encourage the development of drugs to help people with rare diseases. Drug companies…
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…
By Rachel Whetstone from In The Cloud Copy When Helisabed Romano was pregnant with her daughter, she got some bad news. The the developing baby had spina bifida, a defect…
This is part two of the story, click here to read part one After searching for a new neurologist, we found one only 30 mins away that was willing…
Seizures are a common symptom of Angelman syndrome (AS) and the cause of these seizures was unknown for a long time. This lack of knowledge made it impossible to…
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…
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