Earlier this year, I interviewed Dr. Arthur Suckow, the CEO and co-founder of DTx Pharma, about the innovative RNA therapeutics being developed for Charcot-Marie-Tooth disease type 1A (CMT1A). In…
Given that glioblastoma tumors are capable of making their own blood supply, these tumors are often aggressive and difficult to treat. The current standards-of-care are still relatively ineffective. As…
The Progeria Research Foundation reports that as of March 2023, there are 193 children and young adults living with progeria or progeroid laminopathies in 51 countries across the globe.…
If you’ve ever heard of alpha-synuclein before, you may have heard it described in relation to Parkinson’s disease. Alpha-synuclein clumps in the brain; while doctors are still trying to…
This year, the American Society of Gene & Cell Therapy (ASGCT) held its 26th annual meeting from May 16 through May 20, 2023. In a news release, biotechnology company…
As it currently stands, it can be difficult to diagnose a rare disease. Many patients require multiple specialist visits over years (the average rare disease diagnosis takes 4-5 years,…
Although the 5-year survival rate for pancreatic cancer has slightly risen (from 11% to 12%), there is still much work to be done. Pancreatic cancer can be difficult to treat.…
Did you know that June is considered Dravet Syndrome Awareness Month? Every year, people within the Dravet syndrome community come together to amplify stories, raise awareness, and encourage the general…
Relyvrio was officially approved by the FDA for treating ALS in September 2022. However, according to an article in BioPharma Dive, there was considerable controversy surrounding the clearance of the…
When it comes to finding novel ways to treat or cure cardiovascular diseases, Cereno Scientific is committed to making change. The clinical-stage biotechnology company works in the realm of both…
An announcement by Pfizer was recently published in Biospace outlining the results of BASIS its Phase 3 clinical trial (NCT03938792). The drug being studied, marstacimab, met its primary endpoints…
In late May 2023, clinical-stage biotechnology company AiViva Biopharma Inc. ("AiViva") shared via news release that the company had launched a Phase 1 clinical study evaluating AIV007 for diabetic…
In August 2022, I reported on a story that touched my heart: Lucy Landman's PGAP3 diagnosis. With less than 50 diagnosed cases of PGAP3 worldwide, parents Zach and Geri jumped…
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…
At the very top of the Rensselaer Polytechnic Institute's website reads a bold statement and call to action: "Why not change the world?" As one of the United States' most…
The American Society of Clinical Oncology (ASCO) held its Annual Meeting from June 2-6, 2023. During the meeting, a variety of stakeholders discussed clinical practices, guidelines, trends, and research related…
Céline Dion’s new milestone is her first-ever film, Love Again, which she stars in while living with a rare disease called stiff person syndrome. Céline expressed her wishes that…
We all dream about finding the absolute love of our life: the person that makes us feel warm and supported, the one we can't imagine our world without. For…
Children born with propionic acidemia require life-long treatment and care; without it, this disorder can lead to coma or even death. While there are supportive treatment options, no current…
Currently, there are two FDA-approved treatments for people living with idiopathic pulmonary fibrosis (IPF): Ofev (nintedanib) and Esbriet (pirfenidone). While these therapies are effective in improving lung function, they…
According to a story from Healio, a recent study found that a treatment combination of adalimumab and low dose methotrexate was able to reduce treatment failure by two times in…
810 words (source) vs. 506 words (mine) - 2% match Chemotherapy, radiation, and surgery may all be utilized to help patients to manage nasopharyngeal carcinoma (NPC), a rare and aggressive…
The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely…
In mid-May 2023, Pharmaceutical Technology reported that a new combination treatment had earned Orphan Drug designation. This combo? Obeticholic acid (OCA) and bezafibrate. Together, they represent a potential therapeutic…
According to a story from BioPharma Reporter, the company bluebird bio has recently submitted its Biologics License Application (BLA) to the US Food and Drug Administration (FDA). This application is…
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