A free MDS Patient & Family/Caregiver Forum, sponsored by The MDS Foundation Inc., will be held in Beverly Hills, California on February 2nd. When: Saturday, February 2nd, 2019 Where: Beverly…
According to a story from BioPortfolio, the keto-medical nutrition company Disruptive Nutrition recently announced that the first patient had been enrolled in a research study which will test a unique…
According to a story from Onclive, combination therapies are likely to play an increasing role in the treatment of chronic lymphocytic leukemia. In this video, leukemia treatment experts discuss the…
According to a story from The Conversation, scientists have determined that sex plays a significant role in outcomes when it comes to treating the rare and deadly brain cancer glioblastoma.…
The Right-To-Try Law is a federal legislation which allows terminally ill patients the opportunity to use experimental drugs that don't yet have FDA approval. It's controversial at best, but some…
Merkel cell carcinoma (MCC) is a form of skin cancer. In the United States, about 2,500 people are diagnosed with MCC each year. The condition is treated with chemotherapy, immune…
According to a press release published by Global News Wire, Australian pharmaceutical company Mesoblast Limited has completed recruitment for its phase 3 study of Revascor - their experimental drug to…
According to a story from Eurek Alert!, a team of scientists affiliated with the University of Helsinki have successfully developed a new, more useful Hirschsprung's disease mouse model. This disease…
According to a story from BioPortfolio, the pharmaceutical company Acer Therapeutics, Inc., recently announced the company's New Drug Application (NDA) has been accepted by the US Food and Drug Administration…
Every day the scientific community makes progress in the study of rare disease. Research in the field has advanced exponentially in recent years and we're starting to see more treatments…
According to a story from BioSpace, the biopharmaceutical company Liquidia Technologies, Inc., has recently released interim safety data for its open label Phase 3 clinical trial testing LIQ861. This data…
According to a story from Medical Xpress, a collaborative team of scientists from UT Health San Antonio, the Mayo Clinic, and the Wake Forest School of Medicine, have recently released…
According to a story from king5.com, nine-year-old Taylor Futch's smile has always been able to captivate those around her. Unfortunately, Taylor's smile is often the extent of her ability to…
According to a story from USA Today, two 29 year old rare disease patients, Daru Smith of Chicago and Sarah McPharlin of Grosse Point Woods, Michigan, both received triple organ…
According to a story from Business Wire, Basilea Pharmaceutica, Ltd., a partner of ArQule, Inc., recently released interim results from its Phase 2 clinical trial testing the company's investigational drug…
Happy Thursday! We hope everyone's new year is off to a good start. If you're in need of some inspiration, we're highlighting a story of a woman with CIDP accomplishing…
The Patient Access Network Foundation (PAN) is an organization which works to ensure that patients living with rare, chronic, or life-threatening diseases are not prohibited from receiving treatment due to their…
According to a story from finanznachrichten.de, the biotechnology company Saniona recently announced that its treatment period for adolescent patients in its Phase 2a clinical trial testing Tesomet as a treatment for…
A recent article by NBC News is highlighting a growing concern among parents and professionals alike over the effectiveness of newborn genetic screening. There is a certain place between knowing…
A new clinical trial which will test a two-part combination treatment for Erdheim-Chester disease was recently added to the clinicaltrials.gov website. This trial will be specifically for patients with BRAF…
According to a story from the USC News, a team of doctors with the University of Southern California (USC) ALS Clinic are doing their best to help Shaun Kalpakoff, a…
According to a story from ABC News, Tony Gibson of Tennessee was recently diagnosed with the human form of mad cow disease, which is also known as Creutzfeldt-Jakob disease (CJD).…
According to a story from physiciansweekly.com, a recent report from the Institute for Clinical and Economic Review (ICER) suggests that, pending more data about overall price and longterm success rates,…
According to a story from Medical Xpress, a team of researchers affiliated with the Johns Hopkins Kimmel Cancer Center found some encouraging results when they tested an immune system enhancing…
According to a story from The Chicago Tribune, insufficient supplies of donated organs that could be used for transplant surgeries result in 33 deaths per day in the US. At…
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