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.
Clustered Regularly Interspaced Short Palindromic Repeats is more commonly known as CRISPR. As reported recently in Science News, the CRISPR/Cas9 “molecular scissors” is slated to make its highly anticipated debut…
Continue ReadingCRISPR Gene Editing Promises to Cure 10,000 diseases. Now in Its First Human Clinical Trial. Will it Eventually Deliver?
In 1996, Tim Green made the comment that you cannot go through an NFL season without doing serious damage to your body. In a recent 60 Minutes interview with Steve…
Continue ReadingFormer NFL Star Tim Green has ALS but No Regrets About His Career
According to a recent article published in News NWA, in 2017 Mark Bedwell, of Fort Smith, Arkansas had been told that based upon the rapid progression of his amyotrophic…
Continue ReadingExpectations For This Clinical Trial Were to Slow Down ALS Progression; Instead it Reversed One Man’s Symptoms
The Center for Disease Control (CDC) is urging doctors and researchers to investigate and to collect new data before the disease strikes again. An article in Ars Technica reports…
Continue ReadingMystery: A Paralysis That Occurs in Children Every-Other-Year
Since the 1940s oncologists have been focusing on tumors at the location in the body where the tumors originated. According to a recent FDA press release, this is the third…
Continue ReadingICYMI: With the FDA’s Third Approval for Tissue Agnostic Therapies, Oncologists are Headed to the Next Level in Cancer Treatment
A recent article published in Curetoday highlights not only the many new and successful therapies available to cancer patients but it also illustrates how patients can connect to other…
Continue ReadingThe Impact of Patient Registries on Research and How You Can Join
According to a publication from EurekAlert, a recent study helmed by the Royal Marsden NHS Foundation Trust suggests that female cancer patients tend to live slightly longer following treatment than…
Continue ReadingICYMI: Study Suggests Female Cancer Patients Have Better Outcomes After Treatment, Experience Worse Side Effects
Allergan plc, a leading global pharmaceutical company, and its partner, Editas Medicine, Inc. have been given the green light for a clinical trial designed to treat patients with severe…
Continue ReadingThe World’s First Editing of DNA in Humans Using CRISPR to Treat Leber Congenital Amaurosis
According to a recent article in CheckOrphan, glioblastoma (GBM) is considered to be among the deadliest cancers in the world. Currently, treatment options are surgery, chemotherapy, and radiation. In most cases,…
Continue ReadingGlioblastoma Clinical Trial: The Henry Ford Cancer Institute Enrolls the World’s First Glioblastoma Patient
A study appeared recently in the Orphanet Journal of Rare Diseases reporting that rare diseases affect 350 million people worldwide. The definition of rare disease differs between the United States…
Continue ReadingImproved Guidelines for Patient Advocacy Groups Supporting Rare Diseases and Working with Pharmaceutical Companies
It's common knowledge among researchers who study diseases called toxic proteinopathies that this group of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…
Continue ReadingResearchers Might Have Just Made a Breakthrough Discovery for Treating Rare Proteinopathies
Patient Worthy wishes to share the following funding opportunities with faculty/ biotech having interest in these areas of research. The two grant programs were announced by the Orphan Disease Center…
Continue ReadingAttention Biotech and Stem Cell Researchers: Two Potential Grant Programs!
Popular Science published an article recently about the first time an attempt was made to treat sickle cell anemia using CRISPR technology. The patient, Victoria Gray, age 41, from…
The headline in a recent Washington Post article declared that the FDA claimed Novartis, through its newly-acquired company AveXis, manipulated data in an application for a gene therapy drug. These…
Continue ReadingThe FDA Will Allow the Most Expensive Therapy in the World to Remain on the Market
It's common knowledge among researchers who study diseases called toxic proteinopathies, that this grou of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…
Continue ReadingResearchers are Working to Repair a Small Step In a Pathway Involved in Neurodegenerative Diseases
As reported recently in PhysOrg by NASA, researchers aboard SpaceX CRS-18 cargo flight are focusing on what triggers Parkinson’s disease and multiple sclerosis, both neurodegenerative diseases. Researchers suspect that…
Continue ReadingThis Time a “First-of-its-Kind” Study is “Out of this World” on Board the Space Station
Doug Lindsay ran track in high school. According to an article published by CNN Health, Doug had big plans as a senior at Rockhurst University in Kansas City where he…
Continue ReadingThis Young Man Accurately Diagnosed His Own Disorder But Doctors Did Not Believe His Diagnosis
While the last few years have seen major advances in all areas of the pharmaceutical industry, some of the newest and brightest talents are now focusing on cell and…
Continue ReadingA Brief Discussion of How Cell and Gene Therapies are Being Improved for Future Generations
As set forth in a recent MD Anderson Cancer Center publication, targeted therapy makes it possible to personalize cancer treatment. It is also known as “precision medicine”. For about forty…
Continue ReadingTargeted Therapy and Personalized Cancer Treatment
Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…
Continue ReadingDNA Sequencing Took Years, Now it Takes Hours; As a Result, Researchers Have Discovered The Cause of Four Rare Diseases
Researchers at McGill University celebrated a “first” by creating a mouse model in a dish to demonstrate the effect that mutations in the SLC9A6 gene have on brain cells.…
Continue ReadingChristianson Syndrome: Symptoms Are Known But Therapeutic Options Are Unknown
The number of people in the U.S. who are permanently disabled or who die each year as a result of misdiagnosis is estimated to be over 100,000. EurekAlert recently reported…
Continue ReadingResearch Team Identifies Diagnostic Errors in Three Classes of Disease Associated with a High Death Rate or Permanent Disability
Many patients may not be aware of the vital role they play in medical discoveries, or that without their participation there would be no medical discoveries. CheckOrphan recently presented…
Continue ReadingThe Essential Role of Patients as “Partners” in Clinical Trials
Genomic studies and advances in technology have increased researchers’ knowledge of several genetic syndromes that put people at risk for acute myeloid leukemia (AML). An article that appeared in…
According to a report recently published in the Inter Press Service, since the advent of multidrug therapy in 1982, over sixteen million people have been cured of Hansen’s disease,…
Continue ReadingSince 1982, Over 16 Million People Have Been Cured of Hansen’s Disease Worldwide, But a Major Struggle Remains in Brazil
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