Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.
The New England Journal of Medicine recently carried a report citing data from a Phase 3 clinical trial involving 307 people diagnosed with MSI-H-dMMR metastatic colorectal cancer. MSI-H (high levels…
ASH 2020: CRISPR and Vertex’s Potential Cure for Sickle Cell Disease and More Glimmers of Hope Published: Dec. 7, 2020 BioSpace CRISPR Therapeutics and Vertex Pharmaceuticals presented new data on…
The United States is coping with an obesity epidemic. It is therefore critical to understand the distribution of body fat and its effect on treatment. A recent article in Healio…
Xavier Alford told BBC News how he vividly recalls the day, twelve years ago, when he was told by his doctor that he had a very rare disease. His doctor…
A webinar hosted by CureDuchenne featuring an informative program by Mesentech is scheduled for 4:30pm EST on Thursday, December 10th. The community is invited to join in and learn directly…
Axcella is a biotechnology company that has developed a new system of treating complex diseases. Its lead candidates are products that treat two liver diseases, as well as NASH, which…
Mesentech and CureDuchenne have announced a partnership that includes generous funding from the Charles H. Hood Foundation. The partnership has developed a targeted therapy for reversing bone wastage in boys…
WCVB NewsCenter 5 recently published a news item featuring Nancy and Paul Burke and their three daughters. The Burke family has been living in the shadows of the deadly Sanfilippo…
An article recently appeared in Health News by NPR describing progeria, an extremely rare disease. There are only twenty people in the United States diagnosed with progeria. The disease causes…
Sorreto Therapeutics recently announced through Globe Newswire that it has filed an application for its investigational new drug COVI-DROPS (STI-2099). The study will investigate the safety and efficacy of the…
COVID-19’s tentacles have invaded the New Drug Applications (NDAs) for 2020. A recent article in Fierce Pharma reported that the FDA has been especially vigilant in scrutinizing NDAs this year,…
The latest news from Israeli scientists, published in the Times of Israel, is that researchers at Tel Aviv University have accurately targeted cancerous cells in mice using CRISPR.…
According to a report in BioPharma Dive, Brainstorm Therapeutics, a New York biotechnology company, said that while its NurOwn therapy for amyotrophic lateral sclerosis (ALS) appeared to have a…
A recent article in BioSpace highlighted presentations by Twist Bioscience, a rapidly growing genomic and synthetic biology company. Twist presented its product line at the virtual 2020 Annual Meeting of…
Rhythm Pharmaceuticals, based in Boston, MA, is a biopharmaceutical company focusing on the development of therapies to treat rare genetic diseases that cause obesity. Until now, there have not been…
According to an article in Biospace, CureVac of Boston, MA, with headquarters in Tübingen, Germany, just announced interim results from its Phase 1 study evaluating CVnCoV, a vaccine candidate against…
CymaBay Therapeutics, a biopharmaceutical company based in Newark, CA, develops and provides access to novel therapies with a recent focus on primary biliary cholangitis (PBC). The company is currently developing…
DNA sequencing has become part of our lives - but what is it? For one thing, it is a big business. In 2003, approximately $3billion US found its way into…
Astellas Pharma, headquartered in Northbrook, Illinois presents its Be R/Ready program to promote patient-physician communication as well as other valuable and informative resources. Their program centers around patients with relapsed…
According to a recent article in ScienceAlert, scientists have conducted experiments in mice activating a protein called protrudin that gives damaged nerve fibers the ability to regenerate. Currently, once nerve…
Bryan and Bradford Manning recently gave an interview to the Associated Press. The brothers told the story of their vision loss as a result of a rare degenerative eye disease.…
A recent article in Science News highlighted a discovery by Richard Seifes, M.D. of Baylor College of Medicine. Dr. Seifes has discovered the origin of a liver disease that…
Colorectal Cancer (CRC) is now the third leading cause of cancer-related deaths in the U.S. A report in Foundation Medicine highlights data on the increase in the number of cases…
Blood cancer treatment is undergoing a transformation, according to a recent study in Nature Medicine. In this regard, the Leukemia & Lymphoma Society (LLS) recently published its own account featuring…
A recent article published in Multiple Sclerosis News Today reported that newly identified immune cells prevented the death of injured nerve cells. A study finds that this particular immune cell…
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