Progeria and Atherosclerosis Progeria, also known as Hutchinson-Gilford progeria syndrome (HGPS), is a rare disease affecting just 400 people across the world. It is caused by a mutated LMNA gene.…
Phase 1 Trial A Phase 1 trial by Gamida Cell is examining a new potential treatment option for patients with multiple myeloma and relapsed/refractory non-Hodgkin's lymphoma who have not responded…
According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…
According to a story from dubainewstoday.blogspot.com, the data from a Phase I clinical trial suggests that an experimental drug candidate from the pharmaceutical company Boehringer Ingelheim could substantially improve the…
What is Bronchopulmonary Dysplasia? Bronchopulmonary Dysplasia (BPD) is a diagnosis most commonly given to premature newborns. In children, BPD is also the most common cause of Pulmonary Hypertension (PH). Premature babies often develop…
In November of 2018, England announced that cannabis could be prescribed for medicinal use by specialist physicians when no other treatments proved effective. It's prescription was purely to be decided…
Happy Wednesday Everyone! We're excited for the spring weather coming up! Today, we're highlighting an article on some good news for the rare community in canada, followed by an update…
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…
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,…
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…
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…
A few months ago, Patientworthy editor Kathy Devanny asked me some important questions about the assistance I receive from Patient Access Managers as a person living with the rare disease…
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…
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.…
According to a story from nbcnewyork.com, scientific research on cells was always a challenging task. Trying to study a certain trait used to mean looking at a tissue sample, which…
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,…
According to a story from Korea Biomedical Review, the Sanofi Genzyme Korea, a subsidiary of the pharmaceutical company Sanofi, recently issued an announcement stating that the South Korean Ministry of…
Ontario, Canada is in the process of working to revamp their healthcare system. One of their primary goals is to increase the role that patients and their families/caregivers play in…
According to a story from BioSpace, the drug developer Horizon Pharma plc recently announced the release of topline data from a Phase 3 clinical trial of its experimental drug teprotumumab,…
As much as we try to minimize human error in the healthcare field, it still happens. To decrease the chance of its occurrence we’ve created various policies, rules, and guidelines…
According to a story from news-medical.net, a results of a recent study could help accelerate the development of therapies for liver diseases based on RNA interference (RNAi). This study demonstrated…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is the collective term used to describe ulcerative colitis and Crohn's disease. These conditions cause diarrhea, rectal bleeding, fatigue, weight loss, and rectal bleeding. These…
According to a story from the Breast Cancer Society of Canada, Veronica Dubois, who is currently pursuing a Master of Science in Medical Biophysics, is focusing her research on developing…
Thanks to a grant from Worldwide Cancer Research, as well as support from various other organizations, researchers at UCLA recently uncovered a new, faster, easier, and more personalized way to…
According to a story from The Indian Express, doctors and drug developers are at a crossroads over an innovative form of therapy called fecal microbiota transplants. These transplants are essentially…
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