As originally reported in PR Week; pharma company Takeda has been joined a plethora of rare patients groups to create the 'I am Number 17 ' campaign which features 17…
Young-onset Parkinson's disease is a mystery to medical professionals. The onset of this condition typically occurs in those older than 60, but in this form the onset occurs between age…
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…
Happy Thursday! This week, we're highlighting a story about a research update on the lung microbiome and how it can be used to predict IPF outcomes. Next, we have an…
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…
I've been hearing people buzz about compression socks/stockings for a while, but I've never invested in a pair myself. They're basically what they sound like-- socks or stockings that... compress.…
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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A medication that works for some patients with a rare disease may not be effective for others, even with the same disease. For rare diseases, this is important because sample…
About three million Americans are affected by inflammatory bowel disease. Over the years there have been many improvements to treatment for this condition, but there are still further advances that…
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…
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…
Cancer patients often face adverse effects from their treatment. These effects typically need to be addressed by a doctor, whether they're nausea, rashes, or anything else. The issue is that…
For much of human history, many genetic disorders were accepted as incurable, their rapid onset bound to take patients' lives before they had the chance to be lived. However, the…
In the wake of Martin Luther King Jr. Day, those with rare diseases have vocalized some of the dreams that they have for the healthcare industry. With all of the…
As originally reported in CBC News, Canada is deciding on new parameters to set for medically assisted death, legalized in 2016. Since legalization, 6,700 Canadians have made use of the service,…
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…
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…
According to a story from streetinsider.com, the Joseph Ahmed Foundation and Tyme Technologies, Inc. recently announced that the first patient has been dosed in a phase 2 clinical trial. This…
Happy Thursday! This week, we're highlighting a story about a boy in Texas whose illness sparked concern about bacterial meningitis. Next, we have a piece from PW contributor Rebekah Palmer…
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