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.…
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…
According to a story from BioBuzz, the innovation of a platform for the delivery of gene therapies could help trigger a golden age of development for this new class of…
People often tell me how upbeat and positive I am, despite living with pain from dystonia all day, every day for the last 20 years. I would say this is accurate…
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…
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…
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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.…
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…
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…
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…
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 newsroom.heart.org, one of the most substantial domestic policy achievements of the Obama administration was the passage of the Affordable Care Act (ACA). Since its passage,…
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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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…
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…
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Genentech (Roche Group) announced positive results of its FIREFISH trial in a recent Business Wire news release. The second part of the study evaluated risdiplam’s efficacy on infants ages one…
Being a teenager is difficult. There are plenty of difficulties and awkward things that teens deal with every day. For those who have a rheumatic disease, their lives can…
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