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…
According to a story from IFLScience, the US Food and Drug Administration (FDA) approved a total of 48 novel medicines last year. A further investigation of these drugs reveals that…
Focal segmental glomerulosclerosis (FSGS) is a kidney disease that often results in end-stage renal failure. A kidney transplant is the best way to treat this failure. The issue with this…
According to a story from the Irish Medical Times, Ireland's Health Service Executive (HSE) has recently announced the extension of a reimbursement agreement between the agency and the pharmaceutical company…
People with rare diseases struggle to get treatment. If you have one, you're probably familiar with the statistic that only 95% of them have treatment options. Rare disease treatments have…
According to a story from The Edge Markets, Dr. Lee Boon Chye, Malaysia's Deputy Minister of Health, has recently announced an initiative to develop a National Framework for Rare Disease,…
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…
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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…
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