Nordic Study Links PNH-Related Blood Clots to Higher Risk of Kidney Failure
A new Nordic study has revealed a significant link between blood clots caused by paroxysmal nocturnal hemoglobinuria (PNH) and an increased risk of kidney failure. PNH is a rare, life-threatening…
Continue Reading
Nordic Study Links PNH-Related Blood Clots to Higher Risk of Kidney Failure
Medicaid Launches Innovative Payment Model to Expand Access to Sickle Cell Gene Therapies
The U.S. Medicaid program has taken a pioneering step to ensure that new gene therapies for sickle cell disease (SCD) are accessible to the millions of Americans who rely on…
Continue Reading
Medicaid Launches Innovative Payment Model to Expand Access to Sickle Cell Gene Therapies
Living Through the Unexpected: My Ongoing Journey with Cervical Cancer
Editor's Note: This story was originally authored by Natalie H., a cancer survivor and loving mother of two. They say cancer changes everything. I’ve learned it doesn’t just change you,…
Continue Reading
Living Through the Unexpected: My Ongoing Journey with Cervical Cancer
Too Little Too Late: A Mom’s Plea for Improved GACI Screening
Acknowledgement: Patient Worthy is honored to share this story from our friends at GACI Global. To view the original article, please click here. Sharon’s passion for children shines in her…
Continue Reading
Too Little Too Late: A Mom’s Plea for Improved GACI Screening
Gene Therapy Breakthrough Restores Hearing in Deaf Patients
A groundbreaking study has demonstrated that gene therapy can successfully restore hearing in patients with hereditary deafness, offering new hope for individuals affected by genetic forms of hearing loss. The…
Continue Reading
Gene Therapy Breakthrough Restores Hearing in Deaf Patients
ScanCell’s Skin Cancer Vaccine Shows Phase 2 Promise, but Only One Candidate Moves Forward
ScanCell, a biotech firm focused on immunotherapies, announced promising results from Phase 2 clinical trials for its skin cancer vaccines, but only one of its two candidates will advance to…
Continue Reading
ScanCell’s Skin Cancer Vaccine Shows Phase 2 Promise, but Only One Candidate Moves Forward
Patient Voices Shape Progress in PNH and Aplastic Anemia Care
A growing focus on patient-reported outcomes (PROs) is transforming the management of Paroxysmal Nocturnal Hemoglobinuria (PNH) and aplastic anemia, two rare and serious blood disorders. According to a recent article…
Continue Reading
Patient Voices Shape Progress in PNH and Aplastic Anemia Care
When defining UMN, the EU must not leave PKU patients behind
Patient Worthy is grateful to provide you this story on behalf of the European Society for Phenylketonuria and Allied Disorders Treated as Phenylketonuria. E.S.PKU is the umbrella organization of about 41…
Continue Reading
When defining UMN, the EU must not leave PKU patients behind
New Neutrophil Modulator Shows Promise for Safer, More Effective Ulcerative Colitis Treatment
A new investigational therapy targeting neutrophils, a type of white blood cell, may offer a safer and more effective option for patients with ulcerative colitis (UC), according to recent research…
Continue Reading
New Neutrophil Modulator Shows Promise for Safer, More Effective Ulcerative Colitis Treatment
Advancing Therapies Propel Paroxysmal Nocturnal Hemoglobinuria Treatment Market Toward Robust Growth by 2034
The global market for treating Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare but serious blood disorder, is poised for significant growth through 2034, according to a recent analysis by DelveInsight reported…
Continue Reading
Advancing Therapies Propel Paroxysmal Nocturnal Hemoglobinuria Treatment Market Toward Robust Growth by 2034
An interview with Johnny’s Mom: A Dravet Syndrome Family Life
Danielle’s baby boy had a long and terrifying seizure on the 4th of July six years ago. The family was at her mother’s home. Both Danielle and her mother are…
Continue Reading
An interview with Johnny’s Mom: A Dravet Syndrome Family Life
Study Shows Dogs Can Detect Parkinson’s Disease by Smell
A new study has found that dogs are capable of detecting Parkinson’s disease by scent, potentially opening the door to earlier and less invasive diagnosis for the neurological disorder. According…
Continue Reading
Study Shows Dogs Can Detect Parkinson’s Disease by Smell
House Panel Criticizes Federal Oversight of Organ Transplant System After Damning Report
A U.S. House of Representatives oversight panel has sharply criticized the Health Resources and Services Administration (HRSA) following the release of a report revealing serious shortcomings in the country’s organ…
Continue Reading
House Panel Criticizes Federal Oversight of Organ Transplant System After Damning Report
FDA Rejects Ultragenyx’s Gene Therapy for Rare Disease, Citing Manufacturing Concerns
Ultragenyx Pharmaceutical, a biotechnology company developing treatments for rare diseases, has received a setback from the U.S. Food and Drug Administration (FDA) regarding its gene therapy candidate, DTX401. According to…
Continue Reading
FDA Rejects Ultragenyx’s Gene Therapy for Rare Disease, Citing Manufacturing Concerns
New Hope for Epidermolysis Bullosa: Tampere University Licenses Promising Drug to Theravia
A significant step forward in the treatment of a rare and painful skin condition has emerged from Finland. According to News Medical Life Sciences, Tampere University has announced the licensing…
Continue Reading
New Hope for Epidermolysis Bullosa: Tampere University Licenses Promising Drug to Theravia
Living with Familial Chylomicronemia Syndrome
I live with a rare, genetic disease called familial chylomicronemia syndrome, or FCS, which prevents my body from breaking down fats and removing triglycerides from my bloodstream. FCS is inherited…
Continue Reading
Living with Familial Chylomicronemia Syndrome
New Study: Extending Imaging Including the Heart Upon Arrival in E.R. Helps Determine Cause of a Stroke
The study, published in the journal The Lancet Neurology and reported by MedicalXpress.com, is the first to demonstrate the urgency of extending imaging that includes the heart. This must be accomplished within…
Continue Reading
New Study: Extending Imaging Including the Heart Upon Arrival in E.R. Helps Determine Cause of a Stroke
A Life Touched by Fragility: Living with Epidermolysis Bullosa
Epidermolysis Bullosa (EB) is a devastating and rare genetic skin disorder often described as having "butterfly skin." For those living with EB, even the gentlest touch can lead to painful…
Continue Reading
A Life Touched by Fragility: Living with Epidermolysis Bullosa
Alyssa’s Story with Generalized Pustular Psoriasis
“I have to be dying”, I said earnestly to my husband for the second time that year, “at the very least, I don’t think there is much more pain I…
Continue Reading
Alyssa’s Story with Generalized Pustular Psoriasis
Why Does Healthcare Feel Like an Obstacle Course for Patients?
This article was originally published on Ask the Patient. Welcome to the inaugural post of “Ask the Patient.” As a cancer survivor who has experienced medical gaslighting, medical bias, negligent…
Continue Reading
Why Does Healthcare Feel Like an Obstacle Course for Patients?
Finding Joy Through Art: How One North Texas Girl Thrives Despite a Rare Skin Condition
In North Texas, a remarkable young girl named Isabella "Izzy" Esquivel has found a unique way to experience happiness and self-expression—through her art. Izzy, now ten years old, was diagnosed…
Continue Reading
Finding Joy Through Art: How One North Texas Girl Thrives Despite a Rare Skin Condition
Promising Progress: ANSELAMIMAB Advances in Phase III Trials for Light Chain Amyloidosis
A new potential treatment for light chain amyloidosis is making significant strides in clinical development. According to a recent update reported by Drugs.com, the phase III clinical program called “CARES”…
Continue Reading
Promising Progress: ANSELAMIMAB Advances in Phase III Trials for Light Chain Amyloidosis
FDA Halts Sarepta’s ELEVIDYS Distribution and Clinical Trials After Three Deaths Linked to Gene Therapy
On July 18, 2025, the U.S. Food and Drug Administration (FDA) took decisive action against Sarepta Therapeutics following the deaths of three individuals who had received the company’s gene therapy…
Continue Reading
FDA Halts Sarepta’s ELEVIDYS Distribution and Clinical Trials After Three Deaths Linked to Gene Therapy
Oliver’s Story with SynGAP
Patient Worthy is honored to share Oliver's story on behalf of SynGAP Research Fund. SRF is a global group of families committed to accelerating the science to cure SYNGAP1 &…
Continue Reading
Oliver’s Story with SynGAP
- Go to the previous page
- 1
- …
- 12
- 13
- 14
- 15
- 16
- 17
- 18
- …
- 24
- Go to the next page
