Erika Baker and her family have been dealing with the impact of adrenoleukodystrophy (ALD) for generations. The disease has impacted several of her family members, including her uncle, her cousins,…
According to a story from Morningstar, the company Alexion Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has agreed to review their Biologics Licensing Application for their…
According to a story from Business Wire, the biopharmaceutical company Onspira Therapeutics recently announced that its investigational therapy OSP-101 was recently granted Orphan Drug designation by the US Food and…
The US Food and Drug Administration (FDA) has released a statement outlining its plans to promote patient voices in the development and regulation of medical products. You can find more…
According to a story from USA Today, despite numerous warning letters sent from the US Food and Drug Administration to the offices of doctors and other medical professionals across the…
Researchers have found that an artificial intelligence (AI) system is able to recommend patient referrals for over fifty eye conditions as accurately as world-leading doctors. The project is part of…
According to a story from Deseret News, sarcoma patients at the Huntsman Cancer Institute are planting sunflowers as a way to spread awareness about sarcoma, a rare and aggressive form…
The United States FDA has approved the first generic version of EpiPen and EpiPen Jr (epinephrine). The approval was granted to Teva Pharmaceuticals USA, who will now be able to…
Elmo and friends are here to help! - thanks to the good people of The National Association of Community Health Centers (NACHC)! For those who have never heard of the NACHC,…
On August 16th, 2018, Aretha Franklin, the "Queen of Soul," passed away at age 76. Aretha will be immortalized for generations because of the success of her classic soul ballads…
Gabriel Low, a 17-year-old from Hawaii, just completed a 3,000-mile bike ride across the country. He’s been riding since June 11th when he embarked from Seattle, WA to raise awareness,…
The Beginning My story began in February 2011. On January 10th, 2011, my husband and I were involved in an accident in which we slid off the side of a bridge 10 feet face…
In January-March of 2015, I was the healthiest I’d ever been... or so I thought. I worked out, drank plenty of water, had a great lifestyle and meal plan. In…
Our partner, the MDS Foundation, is sponsoring another free event for patients and caregivers. Event Summary: Whether you are a newly diagnosed patient, a long-term survivor, or a caregiver this…
The survival rates for infants and children with brain and spinal cord cancer has improved significantly over the past decades. However, a study published online in early August of…
According to a story from Live Science, a species of tick that is originally found in Asia has begun to appear along the East Coast of the US. The parasite…
Calling all members of the GNE Myopathy community! The Neuromuscular Disease Foundation (NDF) is hosting its 5th Annual Symposium on GNE Myopathy this August 30-31st at the University of California, Los Angeles. For…
According to DAIC, Akcea Therapeutics Inc., has recently announced their publication of results from their study analyzing patient-to-patient connectivity towards the management of the rare disease familial chylomicronemia syndrome (FCS).…
According to a story from stuff.co.nz, a baby from Auckland has been diagnosed with two extremely rare disorders that only occur in one in every 10 million people. Her name…
A recent study into Periodic Fever, Aphtous stomatitis, Pharyngitis and Adenitis (PFAPA) syndrome investigated the impact of the condition on children and found that is was associated with a reduction…
Gemphire Therapeutics Inc. has been recommended to stop a clinical trial by the Data and Safety Monitoring Board at Emory University School of Medicine. The clinical trial, which ran into…
Let's take a moment during these dog days of summer to recognize the SMA community during SMA awareness month! Spinal muscular atrophy (SMA) is a rare genetic disorder that causes…
According to a story from Newswise, an organization called the National Comprehensive Cancer Network (NCCN) has recently released treatment guidelines for a rare type of cancer that can affect women…
According to a story from Kaiser Health News, many patients that are dealing with chronic pain feel stuck in the debate over opioids in the US. As the ongoing crisis…
At the 2018 Aplastic Anemia and MDS International Foundation’s (AAMDSIF) International Bone Marrow Failure Disease Scientific Symposium of March 2018, Dr. David Margolis from the Medical College of Wisconsin, and…
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