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.
Lisa Stockman Mauriello of Summit, New Jersey, is not alone in her struggle to gain access to the company’s experimental ALS drug, tofersen. In fact, according to a recent article…
NORD (the National Organization for Rare Disorders), through PR Newswire, recently announced the results of a new study that reveals the total number of generics, orphan products, and biosimilars currently…
For years, scientists have known the mechanisms underlying normal, as well as the disease-causing (pathogenic) versions, of prions. But according to a recent article published in Sci-Tech Daily, no one…
Marian McGlockin was eighteen months old when she was diagnosed with Niemann-Pick Type C disease, a rare and fatal disorder. Marian could barely speak and was unable to walk. According…
No longer the new player on the team, next-generation sequencing (NGS) is well established as having an influence in clinical care. In recent years, NGS has been responsible for many…
Emflaza (deflazacort), a corticosteroid, was originally approved by the FDA in 2017 to treat Duchenne muscular dystrophy (DMD) in patients five years or older. This approval was expanded in 2019…
The majority of Parkinson’s patients treated with Levodopa experience relief from the major motor effects of Parkinson’s. However, according to a recent article in Biospace, after prolonged use of Levodopa…
According to a recent article in Fierce Biotech, most cancers progress through constant cell division. Scientists at Vanderbilt University are determined to find the reason for this mysterious cell division.…
Most people who are unhappy with the price of their medication are not aware of drug prices in the truly high-end segment of the market. GoodRx, the prescription drug watchdog,…
Genetec Engineering and Biotechnology News recently carried a story giving credit to Baylor College researchers for finding microbes living in the gut that are associated with specific symptoms of neurological…
We seldom see articles about multiple system atrophy (MSA also called Shy-Drager syndrome). The disease is extremely rare and currently, there is no effective treatment. But according to a recent…
A person’s true feelings are often difficult to express during an interview. When asked a question by the interviewer there is no time to rehearse but only grasp at the…
According to a recent article in the Seattle Cancer Care Alliance, the FDA’s recent approval of Axi-cel (Yescarta) will improve the chances of survival for relapsed or refractory follicular lymphoma.…
Molecular Targeting Technologies (MTTI), a radiopharmaceutical company, announced through Biospace that the FDA has given its approval for MTTI to move forward with a Phase 1 clinical trial to study…
It is estimated that one person in forty to sixty thousand is affected by Fabry disease. Approximately five hundred Canadians are known to have Fabry disease. Newswise recently carried the…
Aljazeera recently published an article that highlights the difficulty several families have had waiting for a rare disease diagnosis. In the majority of cases, when the families finally received the…
A recent article in the journal Nature describes the first draft of human genome analysis and sequencing published in 2001 by the Human Genome Project international consortium. The consortium included…
According to a recent article featured in News5Cleveland.com, there are over seven thousand rare diseases worldwide that affect over thirty million people in the United States. Several days ago, patients…
When a patient is told that they have a life-threatening rare disease and then told that there is no cure, every effort is made to attend to their physical needs.…
The Orange County Guide features doctors’ articles in each issue. Dr. Justin West’s search to cure his son’s rare disease will be familiar to families also in search of…
Crispr is no longer a buzzword. According to a recent article in The Guardian, it has become the ‘molecular scissors’ that will enable scientists to rewrite our genes or as often said,…
During a recent WZDX News interview with Dr. David Bick, HudsonAlpha Faculty Investigator Dr. Bick stated that in Alabama alone, there are approximately four hundred thousand people with rare diseases.…
Billions of dollars have been spent in research over decades, resulting in only two therapies that treat symptoms of Alzheimer’s disease (AD). There are no approved methods that diminish the…
Research by scientists at Cornell University and Weill Cornell centered around the endothelial cells that form a barrier between tissues and vessels. They control the flow of fluid and substances…
Policymakers Must Offer Solutions That Provide Patients with Lower Out-of-Pocket Costs For Critical Medicine In addition to coping with chronic illness, many patients are faced with the challenge of…
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