Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
Have you ever heard of a biomarker? In short, a biomarker, or biological marker, is some sort of objective measure which can tell you something about your health or health…
When Rachel Matthews birthed her twin boys, Aiden and Gavin, she was ecstatic. But just a day or two after Rachel had returned home with her boys, she received a…
There are three subsets of Von Willebrand disease (vWD), a genetic bleeding disorder. Type 3 is considered to be the most severe form of vWD, culminating in intense or severe…
If you follow Major League Baseball (MLB), you’ve probably been inundated with news regarding the current lockout – and concerns over whether the season will begin on time. However, if…
How effective is UPLIZNA on treating attacks associated with neuromyelitis optica spectrum disorder (NMOSD)? According to a news release from biopharmaceutical company Horizon Therapeutics plc ("Horizon"), an analysis highlighted that…
Prior to February 2022, there were no approved treatments for those with cold agglutinin disease (CAD). However, that changed on February 4th, 2022, when the FDA announced that it had…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A “rare” condition is one affecting under…
BRUKINSA (zanubrutinib) is a prescription medication which can be used to treat Waldenstrom macroglobulinemia (WM), mantle cell lymphoma, and marginal cell lymphoma. So far, the treatment has been approved in…
Historically, both osteosarcoma and neuroblastoma have been difficult to treat. Therefore, new treatment options are needed for these cancers, which predominantly affect children and teenagers. According to Medical XPress, a…
Within 48 hours following her son Kylan's birth, Emily Bussenschutt had already visited three separate hospitals searching for a diagnosis. 9Honey explains that Kylan was born with epidermolysis bullosa…
Over the last few years, many researchers have been utilizing organoids as a pathway to learn more about diseases and spur drug development. Organoids may be helpful, moving forward, in…
In the past, many researchers have hypothesized a link between multiple sclerosis (MS) and different viruses or viral infections. While these hypotheses are varied in nature, there has rarely been…
In a first-in-human, dose-escalation Phase 1 clinical trial, researchers are evaluating cosibelimab (CK-301) as a potential therapeutic option for those with metastatic cutaneous squamous cell carcinoma (cSCC). So far, shares Cancer…
In a recent news release, commercial-stage biopharmaceutical company HUTCHMED shared that its investigational therapy HMPL-523 had received Breakthrough Therapy designation from the National Medical Products Administration (NMPA) in China. HMPL-523…
Prior to February 2022, there were no approved disease-modifying therapies for those with pyruvate kinase (PK) deficiency. However, this has recently changed! According to a news release from biopharmaceutical company…
On February 16, 2022, biotechnology company Hillstream BioPharma Inc. (“Hillstream”) shared that its drug candidate HSB-1216 received Orphan Drug designation from the U.S. Food and Drug Administration. This particular designation…
On February 15, 2022, biotechnology company Zealand Pharma A/S (“Zealand”) announced that its Phase 3 1703 clinical trial evaluating dasiglucagon for congenital hyperinsulinism (CHI) had reached full enrollment. Within this…
Researchers are consistently looking for ways to deepen their understanding of various conditions, which not only helps with drug development but with patient outcomes. According to Medical XPress, a research…
Prozac (fluoxetine) is used to treat a variety of mental health conditions including major depressive disorder (MDD), obsessive-compulsive disorder (OCD), bulimia, and premenstrual dysphoric disorder (PMDD), among others. You may…
In Part 1 of our interview with Lorrie and Stewart Altman, we discussed the journey to the Late-onset Tay-Sachs disease (LOTS) diagnosis, how Lorrie and Stewart's outlook shaped their lives,…
In a news release from early February 2022, clinical-stage biotechnology company Eureka Therapeutics, Inc. (“Eureka”) shared that two of its therapies – ET140203 and ECT204 – received Orphan Drug designation…
In late January 2022, biopharmaceutical company BridgeBio Pharma, Inc. ("BridgeBio") shared via news release that the first patient had been dosed in the Phase 1/2 ADventure clinical trial. Within this…
48 years of marriage, 2 children, 2 nephews, 7 grandchildren, and 3 great-nieces. It is clear that Lorrie and Stewart Altman, over their time together, have created a beautiful life.…
According to a relatively recent news release from biopharmaceutical company Maze Therapeutics, the company has begun dosing healthy volunteers in a Phase 1 clinical trial. During the trial, researchers are…
Unfortunately, a pancreatic cancer diagnosis also comes with a poor prognosis. Since many people do not receive a diagnosis until later stages of the cancer, the 5-year survival rate is…
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