Genetic disorders are caused by genetic mutations (changes) in your gene. Over the past decade especially, researchers increasingly explore genome editing options to fix or address genetic diseases at the…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Kristina Robinson, a…
When young Emmett Cordes initially complained of knee pain, his mother attributed it to his active nature. However, as the pain persisted, a visit to the doctor and subsequent…
For the first time ever, people living with severe hemophilia A will have access to a gene therapy solution designed to treat their condition at the source. In a…
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…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Approved for use in 2017, emicizumab—marketed under the brand name Hemlibra—has shown immense benefits in the treatment of hemophilia A. But researchers wanted to better understand how emicizumab, used…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families,…
Shifts in treatment paradigms can improve the lives of countless patients and contribute to better overall outcomes. In a news release from February 23, 2023, Sanofi and international biopharmaceutical…
In a recent press release, the National Hemophilia Foundation announced it has started a new campaign called “Start the Conversation” which will initiate conversations around inheritable blood disorders. Bleeding Disorders…
According to Hemophilia News Today, GENV-HEM, an investigational treatment for patients with hemophilia A and B either with or without clotting factor inhibitors, earned Orphan Drug designation from the FDA.…
From May 11 to 14, 2021, the American Society of Gene & Cell Therapy (ASGCT) Virtual Annual Meeting took place. During the 24th annual meeting, a variety of stakeholders met…
In a news release from May 18, 2021, biopharmaceutical company Catalyst Biosciences, Inc. ("Catalyst") shared that the first patient was dosed in the Phase 1/2 MAA-202 clinical trial. During…
At birth, Don Smith was diagnosed with hemophilia A, a bleeding disorder. At that time, treatment options for the illness were limited. Most patients were not expected to live much…
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…
According to ForexTV, commercial gene therapy company Spark Therapeutics ("Spark") recently announced preliminary data from a Phase 1/2 clinical trial evaluating SPK-8016, an investigational gene therapy, for patients with hemophilia…
On January 10, 2021, biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") shared positive data from its Phase 3 GENEr8-1 clinical trial. The clinical trial is evaluating an investigational gene therapy…
In a press release from early January 2021, genetic medicines company Generation Bio Co. ("Generation Bio") announced data from a variety of studies. In one, the company shared that…
Monica and Josh Poynter have recently adopted a nine-year-old boy, Trey, from China. They felt a connection to the boy as he has hemophilia type A, the same rare bleeding…
Last month, biotechnology company BioMarin sought approval for an investigational gene therapy (valoctocogene roxaparvovec) for patients with hemophilia A. However, their approval was denied. Instead, the company was told that…
According to a story from hemophilia.org, the drug company Ferring Pharmaceuticals issued a recall of the nasal spray drug desmopressin (marketed as Stimate) on July 21st, 2020. This product is…
Last Tuesday, the FDA rejected BioMarin Pharmaceutical's gene therapy, Roctavian, for patients with hemophilia A. According to ABC News, the gene therapy infusion was meant to be a single treatment…
During the 2020 Virtual Congress of the International Society on Thrombosis and Haemostasis (ISTH 2020), which took place from July 12-14, participants discussed the latest in treatments, research, and…
According to a story from theindychannel.com, brothers Elias, age eleven, and Emmett, age seven, were both born with the rare bleeding disorder hemophilia A. However, this has not stopped them…
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