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 Colombian family with more than six thousand living family members was the focus of researchers at several institutes in the U.S. and Columbia. According to an article published by…
Continue ReadingThis Colombian Family is Playing a Critical Role in Alzheimer’s Disease Research
Doctor Leah Kaminsky discusses a new era in healthcare in her recent article that offers hope through new technology. How Can AI Help? AI will be able to predict changes to…
Continue ReadingArtificial Intelligence (AI) May Reveal How Our Bodies Predict Our Future Health
A recent article in the Washington Post tells the story of a newly-approved cystic fibrosis drug that represents thirty years of scientific work and dedication. The defective gene that causes cystic…
Continue ReadingFDA Approved Trikafta: It Has the Potential to Benefit 90 Percent of Cystic Fibrosis Patients
The concept of N-of-1, or personalization of clinical care, originated in the 1980s according to an article in The Scientist. It began with a sixty-five-year-old Ontario man who was…
Continue ReadingN-of-1: Clinical Trials for One Person; A Solution or an Impractical Workload?
490 words 8% vs 1306 words 3% According to a recent Globe Newswire release, Progenics Pharmaceuticals, an oncology company that specializes in artificial intelligence and targeted therapies, announced results for its Phase…
Continue ReadingPhase 3 CONDOR Study of PYL: The Potential To Improve The Outcome of Recurrent Prostate Cancer
According to a recent article in Biospace, Catalyst Pharmaceutical’s CMS-001 Phase 3 study ended with mixed results. The trial was billed as the first placebo-controlled double-blind study testing amifampridine phosphate (brand…
Continue ReadingCMS is so Rare That it Took Four Years to Enroll Twenty People for a Trial
A recent announcement by Atomico, one of Europe’s largest venture capital firms based in London, introduced its new partner Healx. The startup can boast of having the world’s most comprehensive…
Continue ReadingThe AI Revolution in Rare Disease Drug Discovery
A recent article published in Cancer Research UK informs readers that in two out of every one hundred cancers, doctors cannot find the original tumor. It is this primary tumor…
Continue ReadingNew Tests May Find Treatments for Carcinoma of Unknown Primary (CUP)
Results of a study reported in PLOS ONE that was conducted by a team of scientists indicate that the experimental drug mavoglurant improves responsiveness and eye gaze for patients with…
Continue ReadingThe First Clinical Evidence that the Drug Mavoglurant Improves Responsiveness in Fragile X Syndrome (FXS)
Actus Therapeutics, a privately held portfolio company of AskBio, has recently announced through PRWeb, the initiation of patient dosing in a clinical trial testing its investigational gene therapy ACTUS-101. About Pompe…
Continue ReadingFirst Patient to Receive Gene Therapy in a Phase 1/2 Study of ACTUS-101 in Patients with Pompe Disease
Over the years Alzheimer’s patients and their families have seen more than one hundred twenty drugs that were developed for the treatment of Alzheimer's have failed. For a while,…
Continue ReadingAfter 120 Drug Failures, Can this be the Drug to Slow Alzheimer’s Progression?
A guest speaker at the 2019 Rare Disease Forum recently held at the North Carolina Biotechnology Center acknowledged that screening is necessary to justify the need for a particular…
CNN recently covered a story in the publication Nature about a paper explaining base editing (or prime editing). The researchers who created the technology set forth the process of using base…
Continue ReadingNew Prime Editing Has The Potential to Search and Replace up to 89% of Genetic Defects
According to a recent article in Horizon, the World Health Organization estimates that every one couple out of ten is dealing with fertility issues. The long-held assumption has been…
Continue ReadingAs More Women Delay Pregnancy, Physiological Changes Increase
A recent article in Medical Life Science News reports that a recent study has discovered a new mechanism that interacts with an immune cell receptor called Mincle. This receptor regulates…
Continue ReadingStudy Results in a New Mechanism to Treat Ulcerative Colitis and Crohn’s Disease
A recent article in the Science Daily reports on a study that demonstrated in detail how the human immune system located in the brain is altered during certain diseases such…
Continue ReadingScientists Have Created an Entirely New Map of the Brain’s Own Immune System in Humans and Mice
Scientists believe that “it is in our DNA.” According to a recent article in EurekAlert, a five-year study formulated through Open Targets together with the Sanger Institute and their…
Continue ReadingThe Open Targets Initiative Brings Scientists Closer to Identifying the Cause of Autoimmune Diseases
Lately, there have been many newsworthy articles about Novartis and Zolgensma (onasemnogene abeparvovec-xioi). In May 2019 the FDA approved Zolgensma, a one-time treatment for the most severe form of…
Continue ReadingZolgensma for Spinal Muscular Atrophy: From Approval to Present Day
Jessica Walton described the staff at Queen Elizabeth Hospital in King’s Lynn as being in “a bit of a panic” when they thought that she was having a stroke.…
Continue ReadingIdiopathic Intracranial Hypertension: Symptoms are Common but the Disease is Rare
A recent article in Forbes highlights a study on diagnostic performance published in The Lancet Digital Health. The study represents the first review to compare the diagnostic accuracy of deep…
Continue ReadingThe Next Frontier for Artificial Intelligence: Diagnosing Disease and Designing Drugs
Mila Makovec began her life as a normal, healthy infant, and according to a recent article in Science Magazine, she continued along this path until reaching the age of…
Continue ReadingPersonalized Medicine: A Drug Tailored for a Six Year Old With Batten Disease May Serve as a Template for Similar Rare Disorders
Supplies of a Critical Bladder Cancer Drug are Dwindling Bob Field, a 72-year-old New York banking executive, received a call from his urologist’s office canceling his next appointment. According to…
Continue ReadingSupplies of a Critical Bladder Cancer Drug are Dwindling
According to a recent article published by the University of California San Francisco, Pelizaeus-Merzbacher disease (PMD) is a rare neurological disease affecting young boys. PMD fatalities may occur before the child…
Continue ReadingFound: Pelizaeus-Merzbacher Disease, the Cause, and a Potential Cure
Physician and investigator John Wood, MD, Ph.D. of Childrens Hospital of Los Angeles specializes in studying the challenges facing SCD patients. He has currently focused his attention on how oxygen delivery…
Continue ReadingStudies Reveal How Blood Flow and Oxygen Delivery Are Affected by Sickle Cell Disease (SCD)
According to a recent article in SMA News Today, risdiplam (formerly RG7916) is in ongoing studies of patients with spinal muscular atrophy (SMA). The drug is an investigational medicine developed by Genentech…
Continue ReadingClinical Trials Show Promising Results for Risdiplam in Treatment of Spinal Muscular Atrophy Types 1, 2, and 3
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