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.
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…
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The 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…
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ICYMI: 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…
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The 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,…
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Glioblastoma 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…
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Improved 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…
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Researchers 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…
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Attention 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…
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Can CRISPR Successfully Treat Sickle Cell Anemia?
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…
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The 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…
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Researchers 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…
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This 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…
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This 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…
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A 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…
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Targeted 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…
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DNA 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.…
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Christianson 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…
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Research 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…
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The 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…
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The Controversy Surrounding Stem Cell Transplants
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,…
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Since 1982, Over 16 Million People Have Been Cured of Hansen’s Disease Worldwide, But a Major Struggle Remains in Brazil
Tatiana Legkiy was only two months old when she was rushed to a hospital in San Francisco after an echocardiogram showed that her heart was malfunctioning. A recent article in…
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CRISPR Genome Editing and Stem Cell Technology Uncovered the Cause of the Baby’s Heart Disorder
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…
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Mystery: A Paralysis That Occurs in Children Every-Other-Year
After twenty-two years physician Huang Chun-jung finally learned why his vision and hearing have fallen to thirty percent of normalcy, thanks to research conducted by the National Health Research Institutes…
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Doctors Are Unable to Diagnose Many Rare Diseases. Genome Sequencing is Changing That Scenario
We often see reports about the latest superbug in the news. Superbugs affect approximately two million people each year and of those, about 23,000 people will not survive. The…
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A New Threat to Antifungal Drugs That Could Affect Ten Million People
Myelofibrosis (MF) comes under the heading of rare cancer. A recent article in Newswise, University of Utah, describes the disease as the failure of bone marrow to produce normal…
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Huntsman Lab Research Team Discovers that Selinexor May Benefit Myelofibrosis Patients Who have No Other Curative Options