Terrence Merrill shares the challenging emotional realities of raising a child with Sanfilippo syndrome. You can read more background about the Merrill's battle against this rare disease in our article here.…
At the age of 28, Suzy remembers medical students feeling her puffy hands and feet. Her feet were so expanded she had to wear wide shoe sizes. Her fingers were…
When I was diagnosed with Parkinson's Disease (PD) in 2000, I first thought of my three children and how my diagnosis would impact them. How would I tell them? Would I…
12-year-old Cezy Fosca lives in Kilkenny City and suffers from Morquio syndrome. Her family waited for months on approval for her treatment, after she lost access to it. Finally, the…
When people are faced with cancer, their main concerns are understandably treatment and survival. However, many are shocked to discover that when they survive cancer, another sinister blow on their…
A gene therapy called ABO-202 that is being developed to treat Batten disease has just been granted Orphan Drug Designation by the European Medicines Agency (EMA), reports BioPortfolio. This follows…
The phrase “it takes a village” gets tossed around a lot. But in Codicote, that’s what’s happening. Fundraising events and challenges are being held left and right. All of them…
The US Food and Drug Administration (FDA) has just approved the drug Vonvendi for use in adult patients with von Willebrand disease who are undergoing surgery. This is an extension…
Imagine not being able to eat because you cannot swallow your food. Add to that intolerable pain and the inability to sleep at night. These are some of the symptoms…
In order to honor Cystinosis Awareness Day, which takes place on May 7th each year, a man will attempt to walk 57 miles in a single day in order to…
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