As originally reported in the Cambridge Independent; rare disease treatment is full of potential. In part, because there aren’t many. This is not for lack of ability, but for lack…
As originally reported in Medscape; many rare disease patients know the frustration of not having any drugs or treatments available for their disease. 95% of rare disease patients have no…
The Calgary Herald published a story about 2 and 1/2 year old Armando Perez's rare disease Progressive Familial Intrahepatic Cholestasis (PFIC), with has left him unusually itchy. It's so important…
If you are unable to work because of a medical condition, you may qualify for disability benefits from the Social Security Administration (SSA). The SSA oversees two different disability programs, Social Security Disability…
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Tahnee Hamilton was recently interviewed by KSL-TV about her young child, Avery, who was diagnosed less than a year ago with T Cell Acute Lymphoblastic Leukemia. About Veno-Occlusive disease (VOD) Avery…
John Lewis, a longtime United States congressman and civil rights leader who helped organized the historic 1963 March on Washington, announced he's undergoing treatment for stage 4 pancreatic cancer. At…
The EMA has just announced that Danicopan has been accepted into its PRIority MEdicines (PRIME) program. Danicopan is being developed for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition. PRIME…
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…
According to a story from globenewswire.com, the biopharmaceutical company Lexicon Pharmaceuticals has recently announced that it has completed its safety review of the initial cohort for its phase 2 clinical…
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,…
For a long time, Catherine Allen went without a diagnose to explain the myriad of symptoms that she experienced. Doctors dismissed her experiences as common feelings in teenagers, when in…
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CNN reported details of Ed’s illness as related by his fiancée Katy Berteau, who remained at this side continually despite risking her own health. Katy and Ed had planned to…
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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…
By Caitlin Seida from In The Cloud Copy If you asked Sarah Baldwin, age 19 and a sophomore at Syracuse University, about the success of the comic book she helped…
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…
According to a story from Pulmonary Hypertension News, a recent study has determined that cardiopulmonary exercise testing can be an effective method to help identify systemic sclerosis patients that are…
The 27th United European Gastroenterology Week took place in Barcelona, Spain in October, at which some of the leading experts in gastroenterology spoke about their field. One of the…
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