Why Getting Funding for Rare Disease Clinical Trials is so Difficult
Why are rare diseases so difficult to study? Is it just their rarity? Or is it also industry challenges? Unfortunately, industry has a lot to do with it. Clinical Trial…
Why are rare diseases so difficult to study? Is it just their rarity? Or is it also industry challenges? Unfortunately, industry has a lot to do with it. Clinical Trial…
As originally reported in CBC News, the problem wasn't that Tom Salisbury had a cold. The problem was that it wouldn't go away. No rest and patience were enough and antibiotics…
The Boston Globe recently interviewed the principals in a case brought by the Pelletier family against the Boston Children’s Hospital. The article gives details about a family’s charge of…
Clinical trials are an opportunity for patients to try new treatments and medicines that offer the potential to improve or extend their lives. The number of clinical trials have…
The charges against Baby Jace’s parents, Crystal Bryant (age 24) and Jarvis Bryant (age 27), seemed to imply that they were guilty until two doctors took a closer look.…
Abbey Luffman was a 16-year-old from Owego, New York excited about getting her learner's permit. She was also very generous; she didn't even hesitate to check the box for organ…
Obtaining a diagnosis for a rare disease tends to be very difficult. Doctors do not immediately expect that rare conditions are causing one's symptoms; they blame more common conditions first.…
When your child receives a rare diagnosis that needs instant care, you don't have time to contemplate finances and plan out your budget. You just have to act. That was…
Lyme disease is the most common vector-borne disease, and it has become more prominent due to climate change and urban developments spreading further into wooded areas. Treatment is typically effective…
ACADIA Pharmaceuticals, working with Neuren Pharmaceuticals, has recently been granted the Rare Pediatric Disease designation from the FDA for their therapy Trofinetide. This drug is intended for the treatment of…
Rare diseases often go undiagnosed, which leads to a lack of treatment and support for those affected by them. People with acromegaly, a rare hormonal disorder, have called for change.…
Caption: David Fajgenbaum (right) and I pose for a photo a few years ago in Philadelphia. Credit: National Disease Research Interchange, Philadelphia Tomorrow is Rare Disease Day at NIH, marking…
Mantle cell lymphoma is a cancer that often responds well to initial treatment. Because of this, doctors often recommend intense treatment soon after diagnosis. While it is often successful, sometimes…
Clinical trials and healthcare as a whole are being transformed by new technology. It's essential for clinical trial sponsors to be aware of the new options available to them, and…
Alyla Annac When Alyla Annac was just 19 years old her father passed away from heart failure. He had suffered 3 heart attacks and doctors said there was nothing else…
We're no stranger to 'Stranger Things' breakout star Gaten Matarazzo, who captured our attention playing Dustin on the hit show -- but he also captured our hearts when he became…
As originally reported in Mercer Island Reporter, Jill Hawkins's three births went smoothly. Her eldest reached expected milestones and began to walk and talk. Her daughter, born four years later,…
Advances in medicine have been responsible for longer overall survival in patients with neurodegenerative-neuromuscular diseases. According to a recent report in RTMagazine, an increase in the number of patients…
Jennifer Schlegel’s life began earlier than expected with an unanticipated disease. According to an article in The Lantern, Jennifer was born prematurely and diagnosed with spastic diplegic cerebral palsy.…
The Jack McGovern Coats' Disease Foundation, a nonprofit patient organization committed to supporting critical disease research and raising disease awareness, is offering travel grants for recipients to attend the 2020…
Sedation Versus Sleep Sleep and sedation are not synonymous terms, despite the fact that they are often used interchangeably in the hospital setting. The biggest difference? Sleep is always beneficial…
Christine Wright Christine Wright was an active, athletic, healthy mother of three. But in 2007 she started exhibiting strange symptoms that no one could give her an answer to. She…
According to a story from PR Newswire, scientists are making significant progress in researching new treatments for glioblastoma, a rare and deadly brain cancer. A variety of drug companies are…
During the 1990s, researchers had a dream that by 2020, every individual would have access to their own genome. This would aid in their diagnosis and treatment as soon as…
Without understanding the cause of disease it is difficult to understand how to most effectively care for patients. Instead of addressing the underlying cause of disease, physicians are forced to…