Rocket Pharmaceuticals has received the green light from the US Food and Drug Administration (FDA) to resume its pivotal Phase II Danon disease gene therapy trial, according to a recent…
Patient Worthy is excited to share our most recent episode of our podcast Wait, How Do You Spell That? featuring Lisa Batista. Lisa shares her story with us of living with…
ITF Therapeutics has announced the publication of encouraging long-term data supporting the efficacy and safety of givinostat as a treatment for Duchenne muscular dystrophy (DMD), according to a recent article…
A recent study highlighted in Hematology Advisor explores the challenges of monitoring patients with hemophilia A after gene therapy, specifically focusing on the reliability of laboratory assays used to measure…
I was diagnosed with Type-1 narcolepsy when I was 20 years old. At the time, I was living in Germany, dancing as a professional ballet dancer in an opera house.…
The Rady Children’s Institute for Genomic Medicine (RCIGM) has announced a landmark international collaboration with Sidra Medicine in Qatar to launch the BeginNGS® newborn genome sequencing program—marking a significant step…
According to a recent article from JournalFeed, the relationship between type 2 diabetes and metabolic dysfunction-associated steatotic liver disease (MASLD) is increasingly coming to the forefront of clinical care. MASLD,…
A routine evening became pivotal for a Portsmouth family when a bright white reflection appeared in nine-month-old Nora’s left eye under bathroom lights. As was reported by Mirror, what seemed…
Acknowledgement: Patient Worthy is honored to share this story by Erika Stariha, Co-Founder and President of SATB2 Europe. This is Urban, my teenage firstborn. He is joyful. Curious. Full of…
The Alpha-1 Foundation (A1F) has announced a groundbreaking advancement in the fight against Alpha-1 Antitrypsin Deficiency (AATD) with the launch of AlphaDetect, a dedicated non-profit subsidiary focused on revolutionizing the…
The U.S. Food and Drug Administration (FDA) reported the approval of semaglutide injections for metabolic dysfunction-associated steatohepatitis (MASH), marking a major milestone in the fight against this increasingly common and…
ACKNOWLEDGMENT Patient Worthy is grateful to our partner Elephants & Tea for providing the following article by Gina Jackson. Elephants & Tea‘s mission is to help adolescent and young adult…
PTC Therapeutics announced that the US Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the company’s New Drug Application (NDA) for vatiquinone, an investigational therapy intended…
Our youngest son, Jace, is 11 years old with Duchenne Muscular Dystrophy. It is a fatal muscle-wasting disease, and there is currently no cure. We were given the diagnosis on…
In our third installment from our series of reports provided by our partner TREND Community, we are going to be focusing on a different condition over four weeks. This week…
A team of scientists from Rice University, Baylor College of Medicine, and the Jan and Dan Duncan Neurological Research Institute have introduced a significant breakthrough in the study of complex…
Early clinical data from a Phase I/II study at UMass Chan Medical School point to meaningful, measurable benefits from a central nervous system gene therapy for GM2 gangliosidosis, which encompasses…
ACKNOWLEDGEMENT – Patient Worthy is honored to partner with The Fatty Liver Foundation to provide you with this article. The Fatty Liver Foundation aims to improve the diagnosis, treatment & support…
After more than a decade of development, families say time is running out for children with Barth syndrome as the experimental therapy elamipretide faces yet another regulatory delay. Reported by…
A new study recently published in the European journal of Preventive Cardiology and reported by Medical News Today, noted that individuals who added extra steps plus reasonable speed to their…
When baby KJ was diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency—a rare metabolic disorder that can trigger life-threatening ammonia buildup, his family faced a narrow path: strict dietary…
Acknowledgment: This story is sponsored by GSK and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted, and valuable…
The FDA has approved BridgeBio Pharma’s acoramidis, branded as Attruby, for transthyretin amyloidosis cardiomyopathy (ATTR-CM), positioning the oral therapy to compete head-to-head with Pfizer’s tafamidis franchise (Vyndamax/Vyndaqel/Vynmac). According to PharmaPhorum.com,…
A study co-led by researchers at The Hospital for Sick Children (SickKids) and the University of Toronto’s Temerty Faculty of Medicine sheds light on why some very-high-risk childhood brain tumors…
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an infiltrative heart disease driven by protein instability. Normally a tetramer, transthyretin can dissociate into monomers that misfold, aggregate, and deposit as amyloid fibrils within…
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