I was laying on the cold, hardwood floor again after drinking phospho cysteamine(the earlier form of Cystagon, treatment for Cystinosis). My stomach was roiling and I was overheating from the…
In the ultimate feat of recycling, scientists at UCLA and the Howard Hughes Medical Institute have re-purposed the LeXis gene as a cholesterol-lowering agent. A recent press release issued by…
Welcome back Patient Worthians! We are back this week from the 2017 Global Genes Rare Advocacy Summit. Did you attend? If so, what did you learn? Let us know here. This…
The love of creating radio is something that can bring people together, according to the Riverine Herald. Whether it’s the perfect mix of melodies or the desire to connect listeners…
How does one raise awareness? Shouting on the street corner might work for a few hundred, or maybe even a few thousand, depending on how busy the corner is that…
When my son was eight years old he joined the swim team. For two hours a day, five days a week, he splashed around the pool mastering the Australian Crawl…
The Pulmonary Fibrosis Foundation (PFF) is hosting their biennial summit this November! Pulmonary fibrosis covers 200 different conditions that include sarcoidosis, IPF, scleroderma and other rare diseases. The conditions are characterized by…
Over the years, I've talked to dozens of people with Parkinson's disease (PD). They come from all socio-economic backgrounds. Rich and poor. Male and female. But the one thing they…
The Cystic Fibrosis Foundation (CFF) is hosting the North American Cystic Fibrosis Conference (NACFC) in Indianapolis, Indiana. The CFF is an amazing non-profit that aims to cure cystic fibrosis while…
POW! BANG! KAPOW! On a typical day, Thor, Batman and Wonder Woman focus on purging the world of archenemies that threaten our very peace and prosperity. And the way things…
There is a long-standing debate on the ethical ramifications of testing medical procedures and drug treatments on animals. One side argues that nothing can replace the valuable information gathered from…
If you missed the 2017 Global Genes Patient Summit's breakout session Strengthening your Care & Support Team, Home Team Advantage: How to Arrange for Palliative Care, we're here to fill…
Scratchy jeans. No. 2 pencils. Trapper Binders. Squeaky shoes. Floor polish. Helpful bus drivers. Smiling teachers. Ah, the scents, sights and sounds of the first day of school. I loved…
Scientists at the University of Georgia have identified a rare type of glycosylation (more on what that is in a second) which plays a major role in the function of…
Causation vs. correlation. What does that even mean? If you ask your friend who majored in philosophy for a semester-and-a-half in college, you’ll probably get an explanation that sounds more…
Good news for patients suffering from neurological disorders that may have taken away their ability to walk. Multiple sclerosis, cerebral palsy, stroke and other diseases can often lead to paralysis.…
If you or someone you love has bronchiectasis or COPD, you should be aware of the risk of alpha-1 antitrypsin deficiency, or A1ATD. Alpha-1 antitrypsin disorder is an inherited condition…
Pull out those calendars! If you’ve been searching for an opportunity to connect with others living with Waldenstrom’s macroglobulinemia (WM), or with those who help care for these individuals—this may…
Announcement is via the Child Neurology Foundation, republished here with permission: NOMINATIONS NOW ACCEPTED FOR: IS HOPE AND IS HEROES AWARDS The Infantile Spasms Action Network will once again sponsor Infantile…
Everyone knows that businesses compete. If two businesses are in the same market, they have to compete, right? If you choose to buy a Ford, then that means you didn’t…
Nearly 100 million Americans live with chronic pain. That's a world of hurt. Chronic pain is defined as persistent pain that lasts more than three months. According to Johns Hopkins…
Amicus Therapeutics is an American therapeutics company based in Cranbury, New Jersey. They're new groundbreaking treatment announcement, however, has very little to do with America, or even New Jersey for that…
It’s fair to say that many (if not most) Americans are inseparable from their smart phones, and that a device that was pure sci-fi 25 or 30 years ago is…
Most people are familiar with the old adage, “The squeaky wheel gets the grease.” My grandmother used to go a bit further by pointing out, “But the greased wheel that…
Just days before Christmas 2016, the FDA gave a present to some of the children in the United States—they approved the first treatment ever to treat spinal muscular atrophy (SMA).…
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