According to an article from Drugs.com, MediciNova Inc. has announced the first enrollment in its phase 1/2 clinical trial of MN-166, an experimental drug that hopes to target recurrent glioblastoma…
FDNA has created a new type of algorithm which may be able to help diagnose rare genetic conditions like Noonan Syndrome by a simple picture of a person’s face. What’s particularly…
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…
Pliant Therapeutics, Inc., a biotechnology company specializing in developing and commercializing treatments for fibrotic diseases, announced on January 3, 2019 through PRNewswire, that it has initiated a Phase 1 first-in-human…
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…
We tend to daydream about miracle cures for rare and detrimental conditions like ovarian cancer. Sometimes they exist, but often it's more of a battle to find the right combination…
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…
A recent study conducted in Oregon has shown that emergency medical responders may be biased against minority patients. It's the concept of unconscious bias. Discrimination is illegal, but that doesn't…
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…
"Beyond research motivation, I want to give a voice to the disease to help researchers empathize and further understand who all their hard work is going toward." These are words from…
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…
According to a story from drugs.com, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced that is had recently completed a end of Phase 2 meeting with officials from the US…
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…
Prader-Willi Syndrome (PWS) is caused by deleted genes on chromosome 15. This results in improper signaling of satiety, leading to obesity. Individuals with the condition can suffer from respiratory/cardiac disease, gastric…
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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