According to a story from apnews.com, President Trump signed the "Right to Try" bill passed by Congress into law on Wednesday, May 30th, 2018. During the signing ceremony, he was…
Precision medicine, also known as personalized medication, is now surfacing more than ever, but could not have happened without the years of research and billions of dollars in funding to…
Research into possible facial creams containing the medicine rapamycin have found that they are effective for reducing facial tumours in people with tuberous sclerosis complex. The full article can be…
According to a story from the Daily Herald, Kevin Barnes, a man from Wauconda, Illinois, believes that his experience with esophageal cancer helped spur his own spiritual growth. This is…
According to an article from Globe Newswire, the company Shire plc recently presented new data in relation to recombinant human parathyroid hormone (rhPTH) and its relationship to hypoparathyroidism. This information…
“A little dirt never hurt” is an old saying many people probably heard at some point during childhood. The idea, usually expressed after dropping a prized piece of food, suggested…
After years of resistance from the previously dominant Republican party, Virginia lawmakers were successfully able to pass a measure expanding Medicaid to nearly 400,000 more Virginians, according to the Washington…
According to a story from Business Wire, the company CENTOGENE, which is dedicated to researching rare genetic disease, will be partnering with the biotech company Aldeyra Therapeutics, Inc. This partnership…
According to a story from pm360online.com, the U.S. Food and Drug Administration (FDA), has given Priority Review status to eltrombopag (marketed as Promacta) after impressive clinical trial results. Eltrombopag was…
According to a story from Telegraph & Argus, 56 year old Andrew Johnson is getting an innovative new procedure that is offered on the UK's NHS in order to relieve…
According to a story from news-medical.net, Mariona Graupera and Frances Viñals, along with Dr. Antoni Riera-Mestre, announced the completion of research that could lead to a new potential treatment for…
According to a story from news-medical.net, the results of a recent research trial demonstrate that the radiation therapy drug AZEDRA (ultratrace iobenguane I131) is an effective and safe treatment option…
May 15 to June 15 is designated as Tourette Syndrome Awareness Month, a rare neurological disorder that causes involuntary movements or vocalizations known as tics. Examples of these tics include repetitive blinking, clearing…
Spinal muscular atrophy is a genetic disease with life threatening implications. In order to create effective therapies, doctors need the ability to target the root genetic mutation and stop it.…
A Phase II trial of the experimental treatment SurVaxM, under development by the biotechnology company MimiVax LLC, has produced encouraging interim results for treating patients with newly diagnosed glioblastoma. The…
The Boston-based biopharmaceutical company Berg LLC has announced that their experimental drug candidate BPM 31510 (ubidecarenone) has been awarded Orphan Drug designation by the US Food and Drug Administration (FDA)…
The United States Food and Drug Administration (FDA) has granted the drug tafamidis Breakthrough Therapy designation for treating patients who have transthyretin cardiomyopathy. The full article can be read here,…
According to a story from BioPortfolio, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that the Phase 2 trial for its proprietary, investigational product sotatercept. The medication is in development…
The PBCers Biannual Conference recently took place in Houston, Texas. The 2018 PBCers Conference was highly informative with topics ranging from the basics of Primary Biliary Cholangitis, dietary tips, updates…
The United States Food and Drug Administration (FDA) has approved the label update for the medicine Taltz (ixekizumab) 80 mg/mL injection for psoriasis that affects the genital area. You can…
Fresh off of Memorial Day, there is no better documentary to watch right now than HBO's John McCain: For Whom the Bell Tolls. The Arizona senator announced last July that he…
Two organisations, NeuroVive Pharmaceutical AB from Sweden and Yungjin Pharm Corporation based in South Korea, have announced encouraging initial results from a Phase I clinical trial of the experimental medicine…
The companies Centogene and Orphazyme are collaborating on a clinical trial of the experimental small molecule therapy arimoclomol for Gaucher disease. The full article can be read here, at BusinessWire. Gaucher…
A drug called Hemlibra that is currently only approved for treating a subset of patients with haemophilia A has been found to be effective for wider use, reports Hemophilia News…
When others learn that you are the parent of a child with a rare disease, they most likely correctly assume that doctor appointments and hospitals might be involved. Yet few…
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