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…
30 Things About My Rare Invisible Illness You May Not Know By Debra Richardson 1. The illness I live with is: stiff person syndrome & insulin-dependent diabetes. 2. I was…
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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…
More than 20 years ago researchers at Harvard University, led by professor Oliver Pourquié, discovered how the vertebra in chickens first form. Essentially, a 'tick' initiates the formation of a…
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…
AI Therapeutics is a biotechnology company that uses artificial intelligence to match drugs to indications. They do so through the use of their algorithm, Guardian Angel. This algorithm has…
According to a story from Medical Xpress, a recent study that draws on 30 years of maple syrup urine disease patient data has concluded that while significant improvements have been…
According to a story from Medical Xpress, a team of researchers from the medical school at the University of Massachusetts has revealed new information about the nature of neurodegenerative symptoms…
This is part two of the story, click here to read part one After searching for a new neurologist, we found one only 30 mins away that was willing…
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…
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