A PIONEER trial has been conducted in order to evaluate the effects of avapritinib, a drug meant to treat systematic mastocytosis. Researchers want to find a better treatment for…
The European Organization for Research and Treatment of Cancer (EORTC) has been evaluating zotiraciclib, which is a new treatment for glioma. This drug has recently been granted the Orphan…
Jojo, a ten-year-old girl, has become the first person to ever receive a new gene therapy for the treatment of GM1 gangliosidosis. The treatment, called AXO-AAV-GM1, was used in…
Copyscape score: 6% Currently there are only two FDA approved treatments for the treatment of amyotrophic lateral sclerosis (ALS). These treatments are only modestly effective, partly because ALS is not…
A new treatment has been found to improve the life of those with Parkinson's Disease tremors through a minimally invasive procedure that sends pulses of focused ultrasounds to the…
A new clinical trial will be conducted at The Center for Neurology of the Academic Specialist Center (ASC) in tandem with the Karolinska Institute. This study is meant to…
Surufatinib was recently granted Orphan Drug designation by the FDA. This drug was made for the treatment of pancreatic neuroendocrine tumors (PanNETs). It was created by Hutchison China MediTech…
In a meeting with some of the world's brightest medical minds, the importance of personalized treatment for inflammatory bowel disease was discussed. At the 14th Annual Congress of the…
Copyscape score: 2% Brigham and Women's is launching the nation's first ever center to conduct research on primary sclerosing cholangitis, a rare liver disease that affects about 30,000 people in…
Three medications have been found that safely treat refractory status epilepticus, which is a disorder in which seizures continue even after being treated with common medication. Benzodiazepene medications, medications…
A recent meta-analyses of clinical studies and case reports that examined the safety of idursulfase therapy, which is an enzyme therapy, has shown that this form of treatment is…
People who live with rare diseases have found that it is often difficult to get a diagnosis. Doctors look to common issues before they consider diseases that affect an…
A study conducted by researchers at UC San Diego has revealed new and helpful information about non-alcoholic steatohepatitis (NASH). This study, led by Dr. Joseph Witztum, focused on a…
Global Blood Therapeutics was recently granted priority review by the FDA for their new drug Oxbryta. This drug is meant to treat sickle cell disease, and it is the…
Finding the correct doctor for treatment can be difficult, especially if one has a rare disease. Because few doctors specialize in rare diseases, they are often spread across the…
Eva Batista is just three months old, and her parents describe her as an "expressive, smiley, and happy baby." Eva is one of the youngest people to ever be diagnosed…
Narcolepsy is characterized by drowsiness, often during the daytime. This is a symptom that greatly affects the lives of those with narcolepsy, and for a long time no FDA…
The FDA has recently approved of Viking Therapeutics' Investigational New Drug (IND) application, which allows patients to enroll in their clinical trials. In order to qualify for these trials,…
New knowledge of the genes that affect sickle cell disease and beta thalassemia has allowed for advances in treatments for these diseases. Advances in technology have also helped with…
The development of treatments and medicines for diseases can take a long time. These treatments come from many hours of research, studies, trials, and waiting for approval from the…
Unfortunately, there are downsides to a lot of treatments for various diseases, such as price, frequency of doses, and discomfort. Any one of these factors can discourage people from…
East Anglia's Children's Hospices in Norfolk, U.K., has recently opened up its new facilities after renovations. To celebrate this opening, Kate Middleton, the Duchess of Cambridge, visited and spoke…
Copyscape score: 4% A common symptom of Rett Syndrome is apnea, which is a difficulty in breathing. This apnea has been a symptom that has had no treatment for a…
There have been extreme improvements in genetic engineering over the past fifty years since it was pioneered in the 1970s. It has been used for the treatment of various…
Scientists have been studying animals in order to better understand certain diseases for years. In fact, a genetic disorder that causes blindness called Leber's congenital amaurosis was found in…
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