Oncternal Therapeutics is working on TK216, a drug that is the first in its class to treat Ewing sarcoma. They have recently opened enrollment for Phase 1b of their…
By Danielle Bradshaw from In The Cloud Copy Scientific teams at the Centers for Disease Control (CDC), the National Institute of Environmental Health Sciences (NIEHS), and other research centers have…
Dicerna Pharmaceuticals works in ribonucleic interference (RNAi) therapies as treatments of rare diseases. They have recently been granted the orphan drug designation by the European Commission for their drug, DCR-A1AT.…
Happy Holidays, Zebra Community! As we gear up for the holiday season, we're highlighting four articles about managing rare disease during the holidays. We have some new articles about spending…
According to a story from globenewswire.com, the genetic medicines company Homology Medicines, Inc., has recently released promising early data from its phase 1/2 clinical trial. This clinical trial is testing…
New developments in drugs and technology have greatly improved treatment for a variety of diseases, one of them being inflammatory bowel disease. These developments have bettered the lives of…
According to a story from Medscape, an advisory panel from the US Food and Drug Administration (FDA) has recommended the approval of the drug teprotumumab for intravenous administration as a…
Mantle cell lymphoma (MCL) is a very rare form of cancer, and oncologists may only see a few cases of it through the course of their careers. Because of…
According to a story from PR Newswire, the biopharmaceutical company Soligenix, Inc. has announced recently that its phase 3 clinical trial has officially completed enrollment. This trial is testing the…
According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved the drug golodirsen (marketed as Vyondys 53) as a treatment for Duchenne muscular dystrophy…
According to a story from the Star Tribune, being disabled in Minnesota can be a damaging affair. The state's most common method for caring for disabled people are group homes,…
According to a story from prnewswire.com, the biopharmaceutical company InMed Pharmaceuticals, Inc. has recently announced that that company's Clinical Trial Application has been approved in the Netherlands. This approval will…
There is no known cure for sickle cell disease. Treatment is symptomatic and meant to prolong life. While a blood and bone marrow transplant is an option, not everyone…
Two patients at UMass Memorial Medical Center with Tay-Sachs disease received gene therapy to attempt to correct the mutation in the HEXA gene that causes the disease. The patients that…
The holiday season and end of the year/decade is upon us! As schedules slow down and we begin to reflect, I too began to take stock of the experiences that…
Parents are often worried about the health of their children; they do not want anything bad happening to their kids. They constantly look out for signs and symptoms of…
According to a story from Medical Xpress, a recent study has confirmed the status of palmoplantar pustolosis as a rare, orphan disease. A recent study of patients with the disease…
According to a story from crweworld.com, the biopharmaceutical company BeiGene, Ltd. recently released the results of a phase 3 clinical trial. The goal of this trial was the comparison of…
Big development for those in the primary hyperoxaluria type 1 (PH1) community! Alnylam Pharmaceuticals just announced that its Phase 3 study of lumasiran, an investigational therapy for the treatment of primary…
Gareth Crabb is a thoughtful and caring six-year-old boy from Swansea, U.K. He is spending his second Christmas in the hospital this year, as he has anaplastic large cell…
A big step forward for those with amyloidosis! The European Medicines Agency (EMA) granted Pfizer a positive recommendation for the approval of its treatment for amyloidosis, a rare disorder that…
According to a story from BioPortfolio, the drug developer Rocket Pharmaceuticals recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This study is…
According to a story from globenewswire.com, the drug developer Agios Pharmaceuticals, Inc. has recently released a preliminary analysis of phase 2 trial findings that have served to establish proof-of-concept for…
By Caitlin Seida from In The Cloud Copy 2019 was a record year for drug recalls due to safety and quality concerns. It’s also been a record year for the…
By Caitlin Seida from In The Cloud Copy Medical alert dogs can give their companions a newfound sense of independence and freedom. Enter Bill, a golden retriever owned by Laura…
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