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.
Amber’s determination to find a miracle for her son Maxwell is detailed in a recent BuzzFeed article. Maxwell and his twin sister, Riley, were born in March 2017. For…
Continue ReadingHer Baby’s SLC6A1 Genetic Disorder is Currently Untreatable, But Amber Freed is Committed to Finding a Cure
Dr. Marc Bonaca, a cardiologist with the Brigham and Women's Hospital in Boston, recently gave a presentation at the Las Vegas 2019 VIVA conference on trends in vascular disease. As reported in…
Continue ReadingDr. Marc Bonaca Presents Insight Into Future Trends for Cardiovascular Disease and the Need For Teamwork
Wearing a revolutionary-era tricorn hat, doctor Mathew Maurer stood at a lectern in front of an audience of fellow cardiologists in Philadelphia, decrying the price of a new medication that…
Continue ReadingDoctors who helped develop heart drug now balk at $225,000-a-year price
An article in the Hartlepool Mail announced that Lyla O’Donovan, her parents, and her four brothers and sisters will be celebrating Lyla’s seventh birthday on November 28th, 2019 at…
Continue ReadingA Father is Asking for Worldwide Birthday Cards for His Seven-Year-Old Daughter Lyla Who Is Scheduled for her 10th Brain Operation
An article appeared recently in BioNews announcing the first-ever approval of an RNA-based therapy for use on the British National Health Service. The name of the drug is Patisiran…
Continue ReadingICYMI: hATTR Amyloidosis was Untreatable Until a Gene Silencing Drug was Approved in the UK
The Golden Age of Health and Wellness is Almost Here A recent article in the Vancouver Weekly News describes the euphoria that accompanied the official declaration of the completion…
Continue ReadingICYMI: The Golden Age of Health and Wellness is Almost Here
The family of a little girl who is battling health problems after suffering a brain tumour are appealing for people to make her birthday extra special by sending her cards…
Continue ReadingHartlepool dad’s worldwide birthday card appeal for daughter Lyla O’Donovan, who is facing tenth brain operation
Teamwork prescribed to tackle major demographic trends in vascular disease 13th November 2019 207 Marc Bonaca addresses delegates at VIVA 2019 “Caring for vascular patients is going to take a…
Continue Reading“Do not be an island”: Teamwork prescribed to tackle major demographic trends in vascular disease
“Myasthenia” means “muscular weakness”. According to a recent article in The Star, studies in Asia indicate that there are approximately five new cases of myasthenia gravis (MG) reported each…
Continue ReadingEverything You Want to Know About Myasthenia Gravis
Marten Devlieger’s sister Karen, a cystic fibrosis patient, died of the disease at the age of thirty-three. According to a recent article in CTV News Canada, Marten was also diagnosed…
Continue ReadingA Call for Help From A Cystic Fibrosis Patient Directed at the Canadian Government
About one to three million people in the United States are living with postural orthostatic tachycardia syndrome (POTS). The disease affects one in one hundred teens and is more common…
Continue ReadingPostural Orthostatic Tachycardia Syndrome (POTS): An Underdiagnosed Condition That Doctors Said Was All in Her Head
An article by Dr. Syavra Tipirneni, dermatologist, recently appeared in BioSpectrum News. Dr. Tipirneni points out that we often succumb to our desire for perfect skin without the realization…
Continue ReadingDermatologists Urge Caution When Using Skin Creams
Stéphanie Tomé is an investigator at the Sorbonne Université in Paris, France. A recent article in PacBio describes Tomé’s research into a disease that becomes progressively worse with each generation.…
Continue ReadingUsing PacBio SMRT Sequencing in Myotonic Dystrophy Type 1 and Other Rare Disorders
LETHBRIDGE — A Taber, Alta., man is calling on government to take action and help grant Canadians with cystic fibrosis better access to breakthrough treatments. Marten Devlieger was diagnosed with…
Continue ReadingCanadians with cystic fibrosis being denied access to life-changing drug, Taber man says
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?
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