It’s no secret that being a part of the chronic illness and/or rare disease community can sometimes be lonely and isolating. Many people, while empathetic to the challenges faced within…
According to a news release from biotechnology company Sound Pharmaceuticals, enrollment has begun in the Phase 3 STOPMD-3 clinical trial. So far, the first patient has been enrolled – with…
1On July 27, 2022, global biopharmaceutical company GSK plc shared some exciting news via news release. The company’s therapy Benlysta (belimumab), designed for pediatric patients with lupus nephritis, is now…
The National Organization for Rare Disorders (NORD) recently held its Living Rare, Living Stronger Patient and Family Forum on June 26, 2022. This event took place both virtually and in-person…
Katharina Clark has always wanted to make a difference. For as long as she can remember, Katharina has held a keen interest in the healthcare sector. Before she was diagnosed…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
A hospital in Chennai, India has recently successfully treated a woman with peritoneal cancer. Not only did they remove the tumor, but they correctly diagnosed her cancer after multiple incorrect…
According to a story from Cushing's Disease News, the Cushing's Support and Research Foundation (CSRF) is organizing to support Cushing's Awareness Day, which takes place on April 8th. A day…
Since its inception, life sciences company Solid Biosciences Inc. has been working to develop treatment options for patients with Duchenne muscular dystrophy (DMD). According to a recent press release, the…
Recently, the Janssen Pharmaceutical Companies of Johnson & Johnson reported that they submitted a New Drug Application (NDA) for intravenously administered UPTRAVI (selexipag). The therapy is designed to treat…
A recent article in The American Journal of Managed Care (AJMC) heralds the approval of a third CAR T-cell therapy, called Tecartus. Kite Pharma’s cell-based gene therapy is the first…
According to ONCLive, ripretinib, designed to treat patients with advanced gastrointestinal stroll tumors (GIST), was given Priority Review status in China by the National Medical Products Administration. This status…
A recent article in The American Journal of Managed Care (AJMC) heralds the approval of a third CAR T-cell therapy, called Tecartus. Kite Pharma’s cell-based gene therapy is the first…
Last month, at the American Academy of Dermatology (AAD) Virtual Annual Meeting, Dr. Dedee F. Murrell, MD, explained the efficacy of rilzabrutinib for treating pemphigus. According to MDedge, many…
In a recent webinar hosted by the National Organization for Rare Disorders (NORD), a number of speakers discussed an up and coming project spearheaded by the organization: the Rare Disease…
In a recent press release, biopharmaceutical company Fortress Biotech announced that their study regarding ATP7A targeted next generation DNA sequencing for Menkes disease was published in Molecular Genetics and Metabolism…
In a recent webinar hosted by the International Waldenström's Macroglobulinemia Foundation (IWMF) and held on July 22nd, 2020, Dr. Jeffery Matous from the Colorado Blood Cancer Institute gave an in-depth…
According to a story from Medscape, the US Food and Drug Administration (FDA) has recently approved a new oral solution as a treatment for symptoms associated with narcolepsy, a rare…
According to a story from Globe Newswire, the biopharmaceutical company Ovid Therapeutics and the University of Connecticut School of Medicine (UConn) have announced a new license agreement and research collaboration…
In order to better understand rare conditions and diseases, researchers sometimes create registries and biobanks full of resources. Recently, the Solve ME/CFS Initiative (Solve M.E.) announced the launch of…
If you're part of the rare disease community, then you probably are not a stranger to explaining your condition to others. But think about it. Has this ever been…
In a recent press release, Knopp Biosciences announced that their drug therapy KB-3061 was granted Rare Pediatric Disease Designation to treat patients with KCNQ2 epileptic encephalopathy (KCNQ2-EE), a rare…
For a lot of us, self-care is difficult. This is for a lot of reasons, some of which might be guilt, not enough time, putting others before ourselves, not sure…
According to a story from Buzzfeed News, a recent test of an experimental COVID-19 vaccine being developed by Moderna Therapeutics demonstrated that it was able to produce antibodies in all…
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