A recent study published in BMC Rheumatology has found that a combination therapy could have a benefit for individuals diagnosed with diabetes and ankylosing spondylitis (AS), psoriatic arthritis (PsA), or rheumatoid arthritis…
Patient Worthy has recently begun a partnership with the Glanzmann's Research Foundation, a nonprofit patient organization dedicated to spreading awareness about Glanzmann's thrombasthenia and finding a cure. We spoke with…
According to a story from GlobeNewswire, the biopharmaceutical company X4 Pharmaceuticals, Inc. has just announced that the US Food and Drug Administration (FDA) has granted its investigational product candidate mavorixafor…
Living with an invisible illness can be incredibly difficult. Those around you do not understand the struggle, and they can even discount your illness. Women's Health talked to three women…
According to a story from Pharma Advancement, Bristol Myers Squibb recently announced encouraging findings from a phase 3 clinical trial. This study was testing its drug ozanimod (marketed as Zeposia)…
Researchers have recently conducted a study using mouse models of Charcot-Marie-Tooth (CMT) disease, titled "CMTM6 Expressed on the Adaxonal Scwann Cells Surface Restricts Axonal Diameters in Peripheral Nerves." It was…
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…
The FDA has recently granted the Fast Track designation to AGLE-102, a treatment for dystrophic epidermolysis bullosa. This designation will allow for the treatment to reach patients at a quicker…
When it comes to rare disease research and drug development, there is one crucial part of the equation: patient input. After all, how can companies ensure that they are meeting…
According to a story from Muscular Dystrophy News Today, a gene therapy in development by the pharma company Pfizer, Inc. was recently given Fast Track designation from the US Food…
Fulcrum Therapeutics is beginning a Phase 1 trial of FTX-6058, a treatment for sickle cell disease (SCD). Researchers will focus on the tolerability, safety, and pharmacokinetics of the medication. The…
The A Million Dreams Gala A Virtual Event This virtual gala is a valuable fundraiser event for KrabbeConnect, a nonprofit organization dedicated to supporting the Krabbe disease patient community and finding a…
For patients with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), post-exertional malaise (PEM) can be extremely debilitating and disruptive. PEM occurs when symptoms worsen or become more severe within 12-48 hours…
According to a story from Targeted Oncology, an experimental combination therapy tested on patients with advanced clear cell renal cell carcinoma (ccRCC) appeared to show potential for efficacy in a…
In late September, GlaxoSmithKline announced FDA approval of its treatment, Nucala, for patients with hypereosinophilic syndrome (HES). The treatment is safe for patients older than 12. Additionally, this approval is…
As written in BioSpace, Genetech has recently presented new two-year data on their spinal muscular atrophy (SMA) treatment, evrysdi. The information was released at the 25th International Annual Congress of…
On September 30, 2020, BioCryst Pharmaceuticals ("BioCryst") announced promising data on their drug candidate BCX9930. The therapy, an orally administered Factor D inhibitor, is designed to treat patients with previously…
The FDA has recently granted two designations, the rare pediatric disease and orphan drug designations, to Spirovant Sciences' gene therapy for cystic fibrosis (CF). SPIRO-2101 will now be able to…
Nate Peters is an air force veteran, and he also lives with ankylosing spondylitis. This means that he knows what it's like to spend a lot of time in the…
Historically, clinical trials have played an immense role in collecting patient data, understanding disease progression, and crafting more targeted treatments. This is especially true in the oncology realm. But according…
Eisai has announced that they will begin a Phase 3 trial, titled MOMENTUM, to evaluate lorcaserin as a treatment for Dravet syndrome. This decision comes after a consultation with the…
The company Lidl GB has just announced a recall for Cologran Stevia tablets. These sweeteners contain aspartame, or E951. Although not harmful for most people, it is essential for this…
A biological marker ("biomarker") is a measurable trait or characteristic that can tell researchers something about your body or health. For example, body mass index (BMI) is a common biomarker…
According to a story from PR Newswire, Chiesi Global Rare Diseases and the biopharmaceutical company Protalix BioTherapeutics, Inc. have recently announced that they have launched an Expanded Access Program (EAP)…
According to a story from BioSpace, the biopharmaceutical company Mereo BioPharma Group recently announced that its investigational therapy setrusumab has earned Rare Pediatric Disease designation from the US Food and…
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