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…
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…
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…
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…
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…
Biopharma.com reported sharp contrasts, with a landmark approval for a rare disease therapy alongside a high-profile trial miss, and a series of corporate moves reflecting market access and financing realities.…
Diagnosing transthyretin amyloid cardiomyopathy (ATTR-CM) is challenging because its symptoms are vague and overlap with many conditions. Providers typically follow a stepwise approach that combines history-taking, targeted testing, and imaging…
In our second 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…
Acknowledgment: This story is sponsored by Astellas Pharma US Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
Novartis has announced encouraging results from two pivotal studies involving its experimental drug, ianalumab, targeting two different autoimmune indications. As reported by BioPharma Dive, the Swiss pharmaceutical company revealed positive data…
Vepdegestrant, an investigational oral selective estrogen receptor degrader (SERD), has reached a critical milestone as the U.S. Food and Drug Administration (FDA) reviews its New Drug Application (NDA) for use…
Millions of social media posts and billions of views have turned TikTok into an influential platform for mental health conversations, but experts warn that using the app to self-diagnose can…
Recent advancements in the treatment of metastatic urothelial cancer (mUC) have dramatically improved patient outcomes, primarily due to the introduction of antibody-drug conjugates (ADCs) and immune checkpoint inhibitors (ICIs). According…
AstraZeneca, a global leader in pharmaceuticals, has taken a significant step into the rare disease arena in South Africa, aiming to improve diagnosis, treatment, and support for patients facing these…
Sanofi has announced a significant regulatory milestone for its investigational therapy SAR446523, a monoclonal antibody targeting GPRC5D, after the U.S. Food and Drug Administration (FDA) granted it orphan drug designation…
Australian athlete Tiana Death has bravely shared her journey after being struck by autoimmune encephalitis, a rare and serious brain disease that upended her life and forced her to relearn…
The U.S. Food and Drug Administration (FDA) has broadened the approval of Empaveli (pegcetacoplan), a complement inhibitor, to include the treatment of two rare and serious kidney diseases: C3 glomerulopathy…
In a significant advancement for cancer treatment, the U.S. Food and Drug Administration (FDA) has granted accelerated approval to Bavencio (avelumab) for the treatment of patients with locally advanced or…
In Chapel Hill, North Carolina, a family’s life changed dramatically when their young twin daughters were diagnosed with Batten disease, a rare and devastating genetic disorder. According to MSN.com, the…
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