According to a story from USA Today, 61 year old Jerry Devries is currently the oldest living patient with a very rare disease: Proteus syndrome. An unusual disease with only…
According to a story from Pulmonary Advisor, the results of a recent analysis led researchers to conclude that systemic anticoagulation therapy can be beneficial for patients with idiopathic pulmonary arterial…
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…
As originally reported in BioSpectrum; the World Health Organization (WHO) has just approved the disease code for CDKL5 Deficiency Disorder (CDD), adding it to the list used worldwide for the…
'What you can't see can't hurt you.' It's a classic and well-intentioned phrase, but we know it to be quite untrue; especially if you ask any one of the millions…
As originally reported in Cystic Fibrosis Life Foundation, Martha has spent her 24 years of life with cystic fibrosis. She was born with the rare disease which means for a…
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…
According to a story from Financial Buzz, the precision oncology company Notable has recently announced that it has opened enrollment for a new observational clinical trial that will focus on…
As originally reported in Global Data, the FDA has just given the final approval for a treatment options for pulmonary arterial hypertension (PAH) called 'Bosentan.' Pulmonary Arterial Hypertension Pulmonary Arterial Hypertension…
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…
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…
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