Merck & Co. announced positive results from two Phase 3 studies of its kidney cancer drug Welireg, signaling potential expansion into earlier stages of disease.
A groundbreaking discovery by an international team led by Shinghua Ding at the University of Missouri has revealed a previously unknown genetic disorder affecting motor
A Wake Forest University School of Medicine study reports that two existing medications (empagliflozin (Jardiance), a diabetes drug, and intranasal insulin) could improve brain health
As noted in a recent article published in Inside Precision Medicine, in 1992 gene therapy was often discussed but seldom practiced. When Jim Wilson MD,
We’re honored to bring you this video, shared with us by Danny Gereg, that details some of his journey living with bladder cancer. Danny has
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
BridgeBio Pharma has marked another significant milestone in rare disease research, announcing a second late-stage clinical trial success—this time with encaleret, a therapy for the
At the 67th American Society of Hematology (ASH) Annual Meeting, Gilead Sciences and its subsidiary Kite showcased a suite of new data underscoring major progress
Research into amyotrophic lateral sclerosis (ALS) is gaining momentum. According to Citeline, early-stage clinical activity surged from 28 new trials in 2023 to 60 in
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
Cowden Syndrome. A name that sounds almost gentle, until you realize it carries generations of uncertainty. It’s caused by changes in the PTEN gene and
Aldeyra Therapeutics, Inc., a biotechnology leader in immune-mediated disease therapies, has announced encouraging results from its Phase 2 clinical trial evaluating ADX-629 in patients with
As reported by drugs.com, at the American College of Rheumatology (ACR) Convergence congress, Novartis presented encouraging results from its Phase III NEPTUNUS-1 and NEPTUNUS-2 trials,
Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company based in Utah, has announced that its investigational drug, ofirnoflast (HT-6184), has received Orphan Drug Designation (ODD) from
In a groundbreaking moment for transplant medicine, Massachusetts General Hospital (MGH) has concluded its second successful xenotransplantation of a genetically modified pig kidney into a
A collaborative team from Arc Institute, Gladstone Institutes, and UCSF has unveiled a groundbreaking epigenetic editing platform that could revolutionize next-generation cell therapies. Published in
Older adults are at heightened risk for severe anaphylaxis but are less likely than younger adults to receive the recommended treatment of epinephrine, according to
International 15q Day is observed annually on November 15 to raise awareness about a group of rare neurodevelopmental disorders caused by genetic changes in the q11–q13 region of chromosome 15 (often referred to as 15q). These disorders include:
• Prader-Willi Syndrome (PWS) – occurs when the paternal copy of chromosome 15 is missing information in this region.
• Angelman Syndrome (AS) – occurs when the maternal copy is missing information.
• Dup15q Syndrome – occurs when there is extra genetic material from this region on either the maternal or paternal chromosome.
Awareness is crucial because these disorders are often misdiagnosed due to their rarity and complexity.
#international15qday #rarediseaseawareness #praderwilli #angelmansyndrome #Dup15q #PatientWorthy
Alpha-Gal Syndrome: Meat Allergy Cases Surge Across the U.S. bit.ly/3JqoN01 Read now at PatientWorthy.com
#PatientWorthy #alpha-GalSyndrome #meatallergy #AGS
A revolutionary new tool may help provide earlier warnings for patients living with ATTR-CM and who are at an elevated risk of stroke. Read more here:
patientworthy.com/2025/11/15/new-tool-may-detect-stroke-risk-in-patients-with-attr-cm/
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