A major genetic research project called the Deciphering Developmental Disorders (DDD) study is helping families to receive diagnoses, and, in some cases, even identifying new disorders. For more information,…
In a recent statement, Scott Gottlieb, the Commissioner of the US Food and Drug Administration, has re-affirmed the organisation’s commitment to modernising drug regulatory pathways. New changes are hoped to…
The problem Synapses in the brain are the connections between neurons that allow them to communicate with one another. In a healthy brain at least, that's how chemical signals are successfully…
According to a story from the Dartmouth Geisel School of Medicine, a group of researchers associated with the school recently published a study that indicated that certain scleroderma patients that…
According to a story from Cure Today, a recent study has found that identifying mutations in patients with myeloproliferative neoplasms may be the key in predicting outcomes, such as disease…
According to a story from mdmag.com, the publication recently conducted an interview with Dr. Eric Ruderman. Eric is a professor at Northwestern University with the Division of Rheumatology. The magazine…
Patients as Partners When you're diagnosed with an illness such as metastatic breast cancer, pancreatic cancer, or another rare disease that has limited treatment options, it's very easy to lose…
New data has been released about the effects of lenabasum, an experimental drug, on patients with systemic sclerosis and dermatomyositis. Corbus, who are developing lenabasum, are planning to present the…
Randy Hilliard, a doctor of psychiatry and University of Michigan professor, awards his survival to the privilege of being so close to a worldwide known cancer center and having the…
The cultural evolution of marijuana hit a new high when Canada recently legalized its recreational use, which also helped opened the door further to the medical exploration of marijuana's effectiveness.…
According to a story from Multiple Sclerosis News Today, Dr. Robert Fox and Dr. Robert Bermel, both of the Cleveland Clinic, recently spoke about new treatment improvements that are needed…
Happy Halloween! As the spookiest day of the year approaches, we're highlighting FDA announcements from the last week and a half we think you should hear about. Check them out…
Last time Patient Worthy wrote about Mila Makovec, she was seven and beginning a cutting-edge experimental treatment for her Batten disease. It’s now nine months later, and Mila is showing…
I live in North Carolina. where we were recently hit with a powerful hurricane that caused a lot of damage. Many trees fell on and around my house. One tree…
For some fortunate individuals, cancer is kind of a foreign concept. They obviously know what it is- they hear cancer stories in passing, they may have donated to a charity…
All things Levi. Such a generally fun-loving little boy, yet such a complex bundle of medical conflagrations. All blended and tied up in a robust package that makes Levi a…
According to a story from pm360online.com, the specialty pharmaceutical company Santhera Pharmaceuticals has recently announced of its Phase Ib/IIa clinical trial that will test its experimental product POL6014. This therapy…
According to a story from Inside Philanthropy, a recent case study was conducted in order to determine the main sources of funding for rare disease research. This broad ranging study…
According to a story from technology.org, The National Center for Advancing Translational Sciences (NCATs) has been hard at work coming up with new methods for engaging with the public and…
According to a story from globenewswire.com, Galapagos NV and Gilead Sciences, Inc., recently announced that two clinical trials which were recently published in The Lancet demonstrated that the experimental treatment filgotinib could…
Two children from a Brooklyn family were born with an ultra rare genetic disease called Canavan disease. Their parents' research revealed that gene therapy could possibly treat the disease. Unfortunately,…
Looking back over everything, it all makes sense now. I have been stuck on this never ending roller-coaster for the past 6 years and I have been pushing hard to…
According to a story from EurekAlert!, a Phase I trial is being developed that will test a unique, three part combination treatment for children with neuroblastoma, a rare form of…
According to a story from abc7.com, two surgeons were part of a crowd of thousands that participated in the Los Angeles Cancer Challenge, which took place at UCLA on Sunday,…
According to a story from BioPortfolio, the biopharmaceutical company Deciphera Pharmaceuticals, Inc., recently announced the release of preliminary data from the company's Phase I clinical trial, which is still in…
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