Happy Thursday! Today, we're highlighting a piece from a PW contributor sharing why it's important for people in the rare disease community to vent sometimes. If you'd like to use…
According to a story from Market Screener, the biotechnology company BioMarin Pharmaceutical Inc. recently announced that the Chinese government has approved the company's drug elosulfase alfa (marketed at Vimizim) as…
According to a story from BioPortfolio, AstraZeneca and Merck have recently announced the results from a phase 3 clinical trial testing olaparib (marketed as LYNPARZA®) as a treatment for BRCA…
According to a story from CBS Minnesota, the Wahlstrom family has been hit hard by a deadly rare illness called Batten disease. Parents Todd and Laura's two children, Drew and…
According to a publication from Scleroderma News, a study recently published in Clinical & Transitional Immunology suggests that certain cytokines (small proteins that play an important role in communication between…
According to a story from BioPortfolio, the gene therapy company bluebird bio, Inc. recently announced that its gene therapy ZYNTEGLO™ has earned conditional marketing authorization in the European Union from…
According to a publication from Biospace, British clinical drug developer Orchard Therapeutics has secured global intellectual property rights to research, manufacture, and market a gene therapy program for the treatment…
According to a story from Market Screener, the drug developer RegeneRx Biopharmaceuticals, Inc. has recently announced that its licensee for RGN-137, an experimental wound healing gel, has begun the dosing…
According to a story from BioSpace, the drug developer Daiichi Sankyo Company Limited has recently announced positive data resulting from a pooled anaylsis of a phase 3 trial and a…
According to a publication from Benzinga, the U.S. Food and Drug Administration has granted Breakthrough Therapy status to Bayer's relapsed follicular lymphoma drug Aliqopa (copanlisib) for the treatment of marginal…
The Problem One in every 12 Canadians will be diagnosed with a rare disease in their lifetime. Two/thirds of these individuals are children and most of these patients experience debilitating…
Sydney Mccallister is a 32-year-old woman diagnosed with spina bifida. Essentially, the condition means that the neural tube has not closed all the way during the first month of embryonic…
.jpg)
Ron Davis and his gene-sequencing technologies were mentioned years ago in The Atlantic alongside Elon Musk (SpaceX) and Jeff Bezos of Amazon fame. Since then, Davis has amassed over…
According to a story from westernmassnews.com, the community of Monson, Massachusetts is coming together to support Jason Harris and his family. Jason is ten years old and lives with Duchenne…
Passage Bio has just recently announced that Krabbe disease will be the focus of their third clinical trial program. Krabbe Disease Krabbe disease is a rare, inherited, neurodegenerative lysosomal storage…
According to a story from statnews.com, one pharmaceutical company is taking advantage of a little known loophole in the Orphan Drug Act to suppress competition and rake in gluttonous profits.…
According to a story from Science Daily, a team of scientists from Johns Hopkins Medicine conducted a review of studies going back nearly 50 years and have concluded that surgery…
According to a publication from Medical Laboratory Observer, a recently published case study following a family with Diamond-Blackfan anemia presents an overview of the condition; and a multi-step approach describing…
According to a story from BioPortfolio, the specialty pharmaceutical company Santhera Pharmaceuticals recently announced that it has submitted its marketing application to the European Medicines Agency (EMA) for its drug…
According to a publication on EurekAlert, the National Institutes of Health recently awarded Dr. Jason Karnes, PharmD, PhD (and assistant professor in the University of Arizona College of Pharmacy) with…
According to a story from brightsurf.com, familial hypercholesterolemia, a genetic condition that causes elevated levels of LDL cholesterol to appear at a young age, often goes undiagnosed. In fact, some…
In a letter to Dr. Norman Sharpless, the acting commissioner of the Food and Drug Administration since April 5, Senator Ted Cruz encouraged the federal agency to expand the number…
.jpg)
Calls to Ban CRISPR A scientist named He Jiankui recently shocked the scientific world with his announcement that he had created twin “CRISPR babies”. The babies, a result of…
According to a story from blogs.biomedcentral.com, the findings from the IPF-PRO Registry, which is a US-based registry for idiopathic pulmonary fibrosis patients, were recently published. Patient registries are a critical…
According to a publication from Oncology Nurse Advisor, a study of 109 patients with myeloproliferative neoplasms (certain types of blood cancers) found that mortality rates for younger patients were similar…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
