SELLAS has just announced that patient screening has begun for a Phase 3 trial for acute myeloid leukemia (AML). The trial is examining the effects of GPS for patients who…
According to a story from IFLScience, the US Food and Drug Administration (FDA) approved a total of 48 novel medicines last year. A further investigation of these drugs reveals that…
Focal segmental glomerulosclerosis (FSGS) is a kidney disease that often results in end-stage renal failure. A kidney transplant is the best way to treat this failure. The issue with this…
According to a story from the Irish Medical Times, Ireland's Health Service Executive (HSE) has recently announced the extension of a reimbursement agreement between the agency and the pharmaceutical company…
People with rare diseases struggle to get treatment. If you have one, you're probably familiar with the statistic that only 95% of them have treatment options. Rare disease treatments have…
According to a story from The Edge Markets, Dr. Lee Boon Chye, Malaysia's Deputy Minister of Health, has recently announced an initiative to develop a National Framework for Rare Disease,…
According to a story from Newswise, a team of scientists and researchers associated with Université de Montréal and CHU Sainte-Justine have recently completed a study that maps a cellular model…
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According to a recent article in the UK Newcastle Chronicle, when doctors gave Leanne Buckley an opportunity to terminate both her pregnancies, Leanne and her husband Michael were emphatic…
According to a story from streetinsider.com, the Joseph Ahmed Foundation and Tyme Technologies, Inc. recently announced that the first patient has been dosed in a phase 2 clinical trial. This…
Surgery has been found to often be the best option towards a cure for a cancerous tumor (resectable) but there are many other tumors that are diagnosed as unresectable…
According to a story from RT, the Lobanov family of Nizhny Novgorod, Russia has been ravaged by Fabry disease. After a bureaucratic quagmire that has gone on for around three…
According to a study on the Wiley Online Library, the genetic factors in pediatric cases of pulmonary arterial hypertension (PAH) are distinct from those of adults. In addition, genetics tends…
By Rachel Whetstone from In The Cloud Copy Justin Stufflebean was 27 years old when he died after being taken to Missouri Western Reception, Diagnostic and Correctional Center (WRDCC). He…
Copyscape score: 7% A new clinical trial is meant to find treatments that can slow or stop the progression of motor neurone disease (MND). This disease causes progressive weakness and…
A New Phase 3 Trial A new phase 3 study will begin soon this year at Stanford University. Abeona Therapeutics had been waiting on IRB approval to begin the trial,…
Body Image In today's world, negative body image is unfortunately very common. Research has shown that this trend occurs in women and men, both as children and adults. It affects…
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According to a recent article in PsychCentral, a rare genetic disorder called 22q11.2 deletion syndrome (22q) is very often misdiagnosed. A study shows that social impairment is the most…
BridgeBio Pharmaceuticals has recently announced that their drug, infigratinib, has received the Fast Track and Orphan Drug designations from the FDA. This drug is meant for the treatment of…
According to a story from globenewswire.com, the biopharmaceutical company SELLAS Life Sciences Group, Inc. just announced that it has started screening participants for its phase 3 clinical trial. The trial…
The discovery of a mutated gene is shedding light on the severity of kidney disease in those with Joubert syndrome. The BSND gene is called a modifier gene, and…
Medical science made some great breakthroughs in 2019 and the worldnewspulse.com shares just a few of the most impressive captured moments for science and real life patients. Here are three…
According to a story from labblog.uofmhealth.org, one of the defining features of Prader-Willi syndrome, a rare disorder, is hyperphagia, which is extreme overeating and obsession with food. While this is…
Prevail Therapeutics is the creator of PR001, which is a gene therapy used for the treatment of Gaucher disease. They expected to begin dosing in the first half of…
The Study: MRx0518-I-003 MRx0518-I-003 is a trial investigating MRx0518 as part of a combination treatment for pancreatic cancer, used with preoperative radiotherapy. The trial will evaluate the therapies safety and…
Sickle Cell Disease Sickle cell disease is a rare blood disorder. It is progressive, debilitating, and sadly can be life-threatening. The genetic defect leads patients' red blood cells to be…
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