According to a story from BioPortfolio, the drug companies bluebird bio, Inc. and the Bristol-Myers Squibb Company have recently announced positive final results for their phase 2 clinical trial. This…
Copyscape score: 9% Deondra Clark was unable to have many of the experiences that other children had. Homecoming, prom, sports, and other childhood activities were not a possibility for her,…
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According to a recently published article in Targeted Oncology, the results of the Phase III CANDOR trial were presented at the 61st ASH annual conference in Orlando, Florida. The…
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A recent article published in Globe Newswire heralded Marinus Pharmaceuticals’ new drug, ganaxolone. The drug has been studied in over 1600 patients and is now being tested in two clinical…
According to a story from EurekAlert! a recent study that was published in the Journal of Huntington's Disease has concluded that while this rare disease community is excited about what the latest…
By Rachel Whetstone from In The Cloud Copy James DeLine works as the village doctor in La Farge, Wisconsin, a rural community with a population of just 750. The village…
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At times there seems to be no happy medium in life. A recent article in Bloomberg claims that the speed at which the FDA is approving newly developed drugs is…
By Caitlin Seida from In The Cloud Copy Jacob Myers was an active 15-year-old until last spring. The honor roll student, who also sang in the school choir and participated…
Personalized Medicine You may have heard of personalized medicine. It's a new wave of healthcare that aims to provide individualized treatment to patients based on their own genetic makeup. The…
According to a story from mHealth Intelligence, the University of Alabama has found success with using telehealth to check in with kidney disease patients that are on dialysis. Now, it…
Mustang Bio, a biopharmaceutical company, has been working with St. Jude Children's Research Hospital to develop MB-107, a lentiviral gene therapy to treat X-linked severe combined immunodeficiency. Mustang Bio…
A drug created by Agios Pharmaceuticals, TIBSOVO, was recently granted the Breakthrough Therapy designation by the FDA for the treatment of relapsed or refractory myelodysplastic syndrome (MDS) with an…
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…
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.…
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…
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…
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…
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