According to a story from biopharmadive.com, the race to a hemophilia A gene therapy continues to tighten following a recent release of early data from Sangamo Therapeutics and Pfizer. The…
Researchers from Brigham and Women's Hospital recently announced a new discovery which could potentially lead to a new multiple sclerosis (MS) therapy. This research has been published in Cell Host & microRNA.…
Therapix Biosciences specializes in cannabinoid pharmaceuticals. Currently, they are working on developing a therapy called tetrahydrocannabinol or THX-110 as a treatment for Tourette syndrome (TS). Additionally, it is being developed…
According to a story from The People's Pharmacy, the US Food and Drug Administration (FDA) has given the green light for the use of generic drugs manufactured in India despite…
According to a story from the Washington Post, rare diseases are a much more widely spread problem than you might think. There are at least 7,000 diseases that are considered…
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…
Some scientists never have the opportunity to meet a single patient in the community of the disease they work their entire lives trying to understand. This can make them feel…
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,…
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…
The medical field is constantly evolving, and rightly so. We should never be complacent with things as they are when it comes to healthcare. There is always more to be…
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…
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…
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…
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…
A recent article in BioPortfolio carried AstraZeneca’s announcement of the FDA’s approval of CALQUENCE® (acalabrutinib) for adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The approval…
Spinal muscular atrophy (SMA) is a rare disorder, but sadly, it is still the most common genetic reason that children die before the age of 2. Not only that, but…
A team of cross-disciplinary researchers at Arizona State University (ASU) is working to develop a software which patients with neurological disorders may easily use to assess their disease progression. Through…
Phase 3 Trial Results from the Phase 3 PREVENT study for neuromyelitis optica spectrum disorder (NMOSD) were extremely positive. 98% of patients in the treatment group did not experience 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…
According to a story from Myeloma Research News, the first patient has been dosed with an experimental allogeneic (donor derived) CAR-T cell therapy called UCARTCS1 as part of a phase…
According to a blog post from Visante, concerns from patient groups and advocates about the safety of a recently introduced tube feeding connector device called ENfit have caused major consternation…
An amendment is being planned for an ongoing Phase 1/2 clinical trial for amyotrophic lateral sclerosis (ALS). This intention has just been announced by the cell therapy company Kadimastem. However,…
It wasn’t until the 1980s that the medical field really started talking about the need to emotionally support patients, especially those with chronic illnesses. This is partly due to the…
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