Ultrasounds and Pain
Imagine being able to turn down the dial on severe pain without taking a single pill or undergoing surgery. Scientists in the UK have just taken a major step toward…
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Ultrasounds and Pain
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Heart Attacks and Your Brain
1. A Physical Smoking Gun in the Heart-Brain Connection When people experience cognitive problems, mood drops, or chronic anxiety after surviving a heart attack, the medical community traditionally viewed it…
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Heart Attacks and Your Brain
How A Rare Genetic Condition May Be Treated by A Simple Vitamin
A recent article from SciTechDaily describes the promising approach instituted at a private, non-profit biomedical research firm in San Francisco that incorporates data science, AI, stem cell biology, CRISPR, and…
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How A Rare Genetic Condition May Be Treated by A Simple Vitamin
Buy Art. Fund Science. Change Lives
Milenia Makani is an abstract artist living in London. She lives with Hypermobile Ehlers-Danlos Syndrome (hEDS), and has launched an international art fundraiser to support medical research through the Ehlers-Danlos…
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Buy Art. Fund Science. Change Lives
The Prevalence of Tick Infections in Pediatric Bipolar Disorders
A recent article by Dr. Rosalie Greenberg explores the possible relationship of infections caused by ticks and psychiatric conditions in children, including PANS or PANDA which are sudden onset. For…
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The Prevalence of Tick Infections in Pediatric Bipolar Disorders
Canadian Biotech Biossil Identifying Potentially Overlooked Drug Candidates
Biossil, based in Toronto and co-founded by Dr. Alexander Mosa (Chair, CSO and co-founder) and Anthony Mouchantaf (CEO and co-founder), is making headway for rare diseases. Biossil’s core model is…
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Canadian Biotech Biossil Identifying Potentially Overlooked Drug Candidates
The PBCers Conference
This, year the PCBers will meet in Indianapolis, Indiana. They will be celebrating 30 years as a community, as well as celebrating a much brighter future with the rare, autoimmune…
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The PBCers Conference
Insights into Gazyva as a Treatment for Lupus/SLE
A recent article from Inside Precision Medicine states: For decades, systemic lupus erythematosus (SLE) has resisted tidy solutions, a shapeshifting autoimmune disease that crosses into different organ systems, flares without…
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Insights into Gazyva as a Treatment for Lupus/SLE
Blood Supply, Malaria, and the Contribution of Grifols Pharma
A New Shield for the Blood Supply: Grifols Earns FDA Approval for Malaria Test Grifols has reached a significant milestone in transfusion medicine with the FDA approval of its Procleix…
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Blood Supply, Malaria, and the Contribution of Grifols Pharma
Regular Yoga Practice Improves Cardiometabolic Health
Yoga is often celebrated for its ability to help people find their "zen," but new research shows it might be doing some heavy lifting for your heart, too! A fresh…
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Regular Yoga Practice Improves Cardiometabolic Health
A Smarter Urine Test for Bladder Cancer
As reported on Inside Precision Medicine, Researchers at Stanford have created a new urine test that helps doctors understand how well bladder cancer treatments are working. Usually, bladder cancer is…
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A Smarter Urine Test for Bladder Cancer
Draft Guidance from FDA Introduces Important Changes to Drug Development for Rare Diseases
On February 23, 2026, FDA issued a Draft Guidance document, called “Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological…
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Draft Guidance from FDA Introduces Important Changes to Drug Development for Rare Diseases
A Promising Approach to Tumor Ablation: Histotripsy
While surgery has long been the standard for removing solid tumors, a revolutionary technique called histotripsy is offering an alternative, as reported on MedPage Today. Histotripsy uses high-intensity, focused ultrasound…
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A Promising Approach to Tumor Ablation: Histotripsy
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The Dravet Syndrome Foundation of Spain’s Annual Meeting 2026
The Dravet Syndrome Foundation of Spain hosted its 2026 Annual Family Meeting on March 20 at the Rafaela Ybarra School in Madrid, bringing together roughly 100 family members, researchers, and specialists.…
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The Dravet Syndrome Foundation of Spain’s Annual Meeting 2026
New Gene Editing Program Aims to Address Root Cause of PKU
For families affected by phenylketonuria (PKU), newborn screening can mean the difference between a healthy future and lifelong complications. PKU is a rare inherited metabolic disorder that leads to toxic…
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New Gene Editing Program Aims to Address Root Cause of PKU
A New Shield Against Cancer: The Lynch Syndrome Vaccine
For people living with Lynch syndrome, the threat of cancer is a constant shadow. This inherited condition affects about one in 300 people, making them up to 80% more likely…
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A New Shield Against Cancer: The Lynch Syndrome Vaccine
CureDuchenne’s 2026 Schedule
CureDuchenne is hosting several free events in 2026 to support families living with Duchenne and Becker muscular dystrophy. These events, called CureDuchenne CARES, will take place across the country. At…
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CureDuchenne’s 2026 Schedule
Netflix Documentary “The Remarkable Life of Ibelin” Honored at 2026 EURORDIS Black Pearl Awards
Brussels — On February 24, 2026, the acclaimed Netflix documentary The Remarkable Life of Ibelin received the Media & Awareness Raising Award at the EURORDIS Black Pearl Awards. The honor…
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Netflix Documentary “The Remarkable Life of Ibelin” Honored at 2026 EURORDIS Black Pearl Awards
Oligodendrocyte‑Targeted Gene Therapy Shows Breakthrough Potential in Canavan Disease
As reported on InsidePrecisionMedicine, a new gene therapy aimed directly at the cells responsible for myelination is showing unprecedented promise for children with Canavan disease, a rare and fatal leukodystrophy.…
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Oligodendrocyte‑Targeted Gene Therapy Shows Breakthrough Potential in Canavan Disease
Patient Worthy Congratulates the Dravet Syndrome Foundation of Spain
Madrid, December 3, 2025 - The research laboratory of the Dravet Syndrome Foundation was one of the initiatives awarded 3,000 Euros in the 11th edition of the Somos Pacientes ("We…
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Patient Worthy Congratulates the Dravet Syndrome Foundation of Spain
How DNA Technology Can Guide Treatment in Muscle Invasive Bladder Cancer
Author's Note: Patient Worthy began following Natera following their presentation at the American College of Medical Genetics conference many years ago. Natera has been known for its advances in prenatal…
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How DNA Technology Can Guide Treatment in Muscle Invasive Bladder Cancer
An Overview on Bladder Cancer
Genetics: a cosmic role of the dice that plays a role in so many medical conditions, including bladder cancer. Mutations in FGFR3, PIK3CA, KDM6A, and TP53 are commonly associated with bladder cancer, but…
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An Overview on Bladder Cancer
“Downton Abbey” Creator Undergoes Transformative Treatment for Tremors
Lord Julian Fellowes, the Oscar-winning creator of Downton Abbey, has undergone a transformative treatment for essential tremor (ET) using Insightec’s magnetic resonance-guided focused ultrasound (MRgFUS) technology. After living with the…
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“Downton Abbey” Creator Undergoes Transformative Treatment for Tremors
Good News for an Ultra-Rare Disease
Each year, Patient Worthy covers the American College of Medical Genetics conference, the leading source of both genetic education for health care professionals and a leading source of new information…
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Good News for an Ultra-Rare Disease
Early Research on Two Tough Cancers
Pancreatic cancer took Alex Trebek, Michael Landon, Ruth Bader Ginsburg, Luciano Pavarotti, Steve Jobs, Patrick Swayze, and my dear friend Amy. Glioblastoma (GBM) took Senators John McCain and Ted Kennedy,…
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Early Research on Two Tough Cancers
