Hope everyone is having a wonderful Wednesday! The weather where we are is freezing this week-- but that's a perfect excuse to sit down with a few of our top…
Danon disease is a rare condition caused by a mutated LAMP-2 gene. This gene has three different forms- LAMP-2A, 2B, and 2C. Depending in which variant is mutated, individuals with…
According to a story from news-medical.net, a recent study has a found that a hidden disease called hemochromatosis could be a major risk factor for several different joint diseases and…
According to a publication from Stat, scientists have high hopes about "virtual repurposing" cutting drug production costs. It could also potentially mean more treatments on the market faster. Virtual Repurposing…
This is week three of this season of The Bachelor on ABC, and week three of a patient-related subject highlighted on the show (Colton's CF foundation, Caelynn's childhood encephalitis)-- so I'm…
Read the first part of Lisa's story here. Lisa’s two direct and indirect bypass surgeries were successful. She now had seven times more blood flow to her brain than she…
According to a story from Biotecnika, Penn Medicine & Inovio Pharmaceuticals Inc. have recently announced that the US Food and Drug Administration (FDA) has signaled its approval for the first…
According to a story from kios.org, firefighters across the country tend to get cancer, including many rare cancers like mesothelioma, at significantly greater rates than the general population. This is…
According to a story from Fabry Disease News, the drug developer Avrobio recently announced that the US Food and Drug Administration (FDA) has given the company's experimental drug AVR-RD-01 Orphan…
What is the Disparity? America is greatly divided by many things and race has always been one of them. Not only are there large social and economic divides among racial…
Canavan disease is a type of leukodystrophy frequently found in the Ashkenazi Jewish population. It's a rare genetic condition which is ultimately fatal. It's all caused by a deficiency of the aspartoacylase…
Emily Sadler is only 15. In March of 2017 she was diagnosed with thyroid cancer. This came as a huge shock to herself and loved ones, especially because thyroid cancer…
The government shutdown hasn't had any startling affects on rare disease research yet, but drug developers have voiced their concern that it could soon begin to hinder the research process.…
Happy Thursday! We hope everyone's week is going well. Today, we're highlighting a contribution from a cystinosis patient on the necessity of third-party medical providers. We're also sharing a story…
Xconomy.com has just released that funds have been raised for a variety of companies investigating therapies for rare conditions. Unfortunately, novel therapies and inventions cost million of dollars to develop. Thankfully,…
Myelodysplastic syndromes (MDS) is the name used to describe a group of stem cell disorders in which the body can't produce an adequate amount of platelets and blood cells. Currently,…
While medical students may go into the profession dwelling on the lives they can save, the inevitable fact of the profession is that there will be times when they…
According to an article recently published by Forbes, one data scientist wants to explain why data science has proven to be slow at identifying particular biomarkers in medical patients. Imran…
At age twenty one, Lisa was about to graduate college and had a promising future career in front of her. All that was left was graduation itself. The week before…
According to a story from PR Newswire, pancreatic cancer rates in the US are increasing by almost two percent each year. This puts the disease in position to become the…
According to a story from New Scientist, a team of scientists at the Institute of Cancer Research in London were able to develop an AI tool that was able to…
According to a story from BioPortfolio, the Bristol-Myers Squibb Company recently announced that the European Commission has recently approved the first combination treatment for renal cell carcinoma available in the…
A free MDS Patient & Family/Caregiver Forum, sponsored by The MDS Foundation Inc., will be held in Beverly Hills, California on February 2nd. When: Saturday, February 2nd, 2019 Where: Beverly…
According to a story from BioPortfolio, the keto-medical nutrition company Disruptive Nutrition recently announced that the first patient had been enrolled in a research study which will test a unique…
According to a story from Onclive, combination therapies are likely to play an increasing role in the treatment of chronic lymphocytic leukemia. In this video, leukemia treatment experts discuss the…
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