First Potential Gene Therapy for Tay-Sachs Disease Produces Positive Results at Three Month Mark

Tay-Sachs disease is a rare, genetic and sadly fatal condition. It's a neurodegenerative disorder caused by an impaired production of the β-Hexosaminidase A enzyme (HEXA). Symptoms of this condition include listlessness, diminishing…

Continue Reading First Potential Gene Therapy for Tay-Sachs Disease Produces Positive Results at Three Month Mark

FDA Regulations and the Critical Differences Between Medical Device Trials and Pharmaceutical Trials

Medical devices include ECG electrodes, bandages, cardiac pacemakers, thermometers, and hemodialysis machines. A comparison of clinical trials shows that medical device trials are somewhat similar to pharmaceutical trials that evaluate drugs,…

Continue Reading FDA Regulations and the Critical Differences Between Medical Device Trials and Pharmaceutical Trials

The Major Ethical Dilemma Behind Rare Disease Drug Costs and a Bioethicist’s Solution

Avraham Steinberg recently spoke at RARE2019, the 2nd International Congress on Advanced Treatments in Rare Diseases. It was held on March 4th and 5th in Vienna, Austria. His talk focused…

Continue Reading The Major Ethical Dilemma Behind Rare Disease Drug Costs and a Bioethicist’s Solution

Phase 2 Clinical Trial for Pulmonary Hypertension has Officially Begun

About Pulmonary Hypertension Pulmonary Hypertension (PH) is a form of high blood pressure which affects the arteries. Primary Pulmonary Hypertension refers to a type of the condition whose origin is unknown. Secondary…

Continue Reading Phase 2 Clinical Trial for Pulmonary Hypertension has Officially Begun

Positive Results from Phase 2 Trial Could Lead to Lower Doses of Corticosteroids for Pemphigus Patients

Pemphigus Pemphigus is a rare autoimmune condition which causes the skin to blister. There are two main forms of the disease called pemphigus foliaceus (PF) and pemphigus vulgaris (PV). Pemphigus…

Continue Reading Positive Results from Phase 2 Trial Could Lead to Lower Doses of Corticosteroids for Pemphigus Patients
Study Shows We Need Better Awareness of Pediatric Onset Multiple Sclerosis to Improve Patient Care
source: pixabay.com

Study Shows We Need Better Awareness of Pediatric Onset Multiple Sclerosis to Improve Patient Care

Multiple Sclerosis (MS) was long believed to be an illness that could only affect adults. In fact, the pediatric form of this disease was not discovered until just over 10…

Continue Reading Study Shows We Need Better Awareness of Pediatric Onset Multiple Sclerosis to Improve Patient Care

Study has Commenced for a New Potential Familial Chylomicronemia Syndrome Treatment

Hypertriglyceridemia Hypertriglyceridemia is the term used to describe abnormally high numbers of triglycerides in the blood. People with Hypertriglyceridemia have an increased risk of heart disease, stroke, and heart attack.…

Continue Reading Study has Commenced for a New Potential Familial Chylomicronemia Syndrome Treatment

Results From the Longest Familial Hypercholesterolemia Study to be Announced at the ACC 2019 Conference

New Research to be Presented In just a few days the American College of Cardiology will host their 68th Annual Scientific Session (ACC.19). It will be held in New Orleans,…

Continue Reading Results From the Longest Familial Hypercholesterolemia Study to be Announced at the ACC 2019 Conference
Close Menu