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…
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…
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…
As originally reported in CBC News, Canada is deciding on new parameters to set for medically assisted death, legalized in 2016. Since legalization, 6,700 Canadians have made use of the service,…
According to a story from IFLScience, the US Food and Drug Administration (FDA) approved a total of 48 novel medicines last year. A further investigation of these drugs reveals that…
Focal segmental glomerulosclerosis (FSGS) is a kidney disease that often results in end-stage renal failure. A kidney transplant is the best way to treat this failure. The issue with this…
People with rare diseases struggle to get treatment. If you have one, you're probably familiar with the statistic that only 95% of them have treatment options. Rare disease treatments have…
According to a story from The Edge Markets, Dr. Lee Boon Chye, Malaysia's Deputy Minister of Health, has recently announced an initiative to develop a National Framework for Rare Disease,…
According to a story from Newswise, a team of scientists and researchers associated with Université de Montréal and CHU Sainte-Justine have recently completed a study that maps a cellular model…
According to a story from streetinsider.com, the Joseph Ahmed Foundation and Tyme Technologies, Inc. recently announced that the first patient has been dosed in a phase 2 clinical trial. This…
Happy Thursday! This week, we're highlighting a story about a boy in Texas whose illness sparked concern about bacterial meningitis. Next, we have a piece from PW contributor Rebekah Palmer…
According to a study on the Wiley Online Library, the genetic factors in pediatric cases of pulmonary arterial hypertension (PAH) are distinct from those of adults. In addition, genetics tends…
By Rachel Whetstone from In The Cloud Copy Justin Stufflebean was 27 years old when he died after being taken to Missouri Western Reception, Diagnostic and Correctional Center (WRDCC). He…
Copyscape score: 7% A new clinical trial is meant to find treatments that can slow or stop the progression of motor neurone disease (MND). This disease causes progressive weakness and…
Body Image In today's world, negative body image is unfortunately very common. Research has shown that this trend occurs in women and men, both as children and adults. It affects…
According to a story from Biospace, the drug company Horizon Therapeutics plc has recently announced that the US Food and Drug Administration (FDA) has approved the company's drug teprotumumab-trbw (marketed…
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As originally reported in Brandpoint, Kailey experienced a haunting tragedy as a child when her sister, Ashley, who was a year younger, experienced crippling symptoms from their shared progressive disease…
As originally reported in CBS News; the flu has been one of humanitys traditional foes. Each season returns with new features and mutations while scientists spend time year speculating what…
According to a video from khou.com, the recent death of a five year old boy in the Houston area from bacterial meningitis has prompted the release of a guideline video…
BridgeBio Pharmaceuticals has recently announced that their drug, infigratinib, has received the Fast Track and Orphan Drug designations from the FDA. This drug is meant for the treatment of…
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