A new precision medicine center, specifically for pediatrics, has launched in Utah. This new center is the result of collaborative efforts from the Intermountain Primary Children's Hospital, Intermountain Precision Genomics,…
By Jack Gerard from In The Cloud Copy. While vaccines are largely considered safe for most of the population, there are some people whose doctors recommend avoiding at least some…
By Jodee Redmond from In The Cloud Copy The results of a study have found that betaine and the active form of folic acid, L-methylfolate, are not effective measures to…
By Rachel Whetstone from In The Cloud Copy For millions of Americans who are affected by rare diseases, hope and health lies in the availability of plasma donations. One use…
The University of Massachusetts Medical School has recently announced preliminary results from their trial for Tay-Sachs disease. This trial is investigating a gene therapy and researchers are excited about it's…
Pulmonary Hypertension Association The Pulmonary Hypertension Association (PHA) is headquartered in Silver Spring, Maryland. It was formed in 1991 by 4 women passionate about finding a cure. Additionally, they hope…
According to a story from BioPortfolio, the biopharmaceutical company Aurinia Pharmaceuticals, Inc. has recently announced positive findings from its phase 3 clinical trial of the experimental drug voclosporin, which was…
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…
For millions of Americans who are affected by rare diseases, hope and health lies in the availability of plasma donations. One use of the donated plasma involves the extraction of…
According to a story from BioPortfolio, Acceleron Pharma and the Bristol-Myers Squibb Company have announced recently that the US Food and Drug Administration (FDA) will review the supplemental Biologics License…
According to a story from Business Wire, the biopharmaceutical company Bristol-Myers Squibb has recently announced that the US Food and Drug Administration (FDA) has granted the company's drug abatacept (marketed…
The iPain Summit 2019 took place from November 14th-16th, 2019. Patient Worthy had the privilege to attend this event and cover it live on social media. This summit was focused…
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…
At the International Pain Foundation's iPain Summit, held this year from November 14th-16th, a wide variety of topics related to the challenges faced by patients with chronic pain were discussed.…
According to a story from Biotech 365, the biopharmaceutical company Avadel Pharmaceuticals plc recently announced that it has completed enrollment for its phase 3 clinical trial. This trial will test…
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…
According to a story from BioPortfolio, the biopharmaceutical company Incyte Corporation has recently announced that the company's New Drug Application (NDA) was recently accepted by the US Food and Drug…
Contact: Britta Vander Linden [email protected] 917.604.6518 30 million Americans need more treatment and diagnostic opportunities https://everylifefoundation.org/rare-disease-community-calls-on-congress-fda-to-enact-life-saving-public-policy-solutions/ (Washington, D.C., December 4, 2019) Hundreds of rare disease advocates from around the country were brought to Washington,…
Getting a diagnosis for a rare disease is hard. Not many people are familiar with the many obscure symptoms and manifestations of these diseases. Because of this, patients with rare…
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A recent article in the Taunton Gazette describes the dilemma faced by the parents of four-year-old Jaxtien Miller who is a metachromatic leukodystrophy patient waiting for a stem cell…
As originally reported in Mirror, when Sian Hopper was in secondary school, she began to get headaches. She attributed them to stress: she had been studying for her GCSE exams,…
A recent study published in the scientific journal PLOS One has determined that patients with the rare disorder primary Sjögren's syndrome are at a greater risk of bladder problems than previously thought.…
Long QT syndrome (LQTS) is a congenital disorder that manifests as a prolonged QT interval on patient electrocardiograms. Those who are affected by LQTS are more likely to experience…
CRISPR/Cas9 technology reached another milestone with encouraging results in two recent trials of CTX001. An article in Pharmaceutical Technology carried an announcement by CRISPR Therapeutics and Vertex Pharmaceuticals about…
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