According to a story from Global Genes, the US Food and Drug Administration (FDA) has said that it will not take the pharmaceutical company Biohaven's recent New Drug Application under…
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 Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In the reality television world, Megan Marx is best known for her time on Bachelor Australia, Bachelor in Paradise, and The Challenge Australia. But Marx is about to be…
Within the United States, the FDA developed the Orphan Drug Act to incentivize companies to develop drugs and biologics intended to treat patients with rare conditions. In this case, "rare"…
GeneDx, an OPKO Health company, has recently launched several genetic tests. Within this group of tests are repeat expansion analysis for Friedreich ataxia, spinocerebellar ataxia (SCA), and other forms of…
As reported in Biospace, Fulcrum Therapeutics has announced it will be submitting an application to the FDA for FTX-6058 as an Investigational New Drug (IND) by the end of 2020…
According to a story from MedicalXpress, the results from a recent study are linking biochemical changes affecting a protein known as CHIP to a diverse array of clinical characteristics that…
According to a story from Bloomberg, the company Cadent Therapeutics recently announced that the company's experimental product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug…
According to a story from Business Wire, the drug developer Cadent Therapeutics recently announced that its investigational drug CAD-1883 has been awarded Orphan Drug Designation from the US Food and…
According to a story from BioSpace, the drug developer Cadent Therapeutics recently announced that its investigational product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug…
According to a story from Kamloops Coffee Talk, Darrin Rein, of Kamloops, British Columbia, was just recently diagnosed with spinocerebellar ataxia, a rare disease that affects mobility and coordination. Despite…
Huntington's Disease & SCAs Huntington's disease (HD) and spinocerebellar ataxia (SCA) are both neurodegenerative diseases caused by the same type of error in the body's DNA. Essentially, the CAG or…
According to a story from Market Screener, the biopharmaceutical company Biohaven Pharmaceutical Holding Company Ltd. recently announced that the first patient has been enrolled in the company's Phase 3 clinical…
Morning, noon, and night Nikki Peterson experiences varying levels of pain, according to an online article in The Root. The pain affects her mood and how she approaches every day.…
Some breathtaking things are happening in the world of sickle cell anemia (SCA) that could potentially impact the lives of nearly 275,000 babies around the world and nearly 100,000 African…
Sickle cell anemia results from a single mutation in the hemoglobin gene. The mutation creates a protein which has an atypical structure. This altered structure throws a wrench in the…
Biohaven Pharmaceutical Holding Company Ltd. announced at the end of last month that the company has completed enrollment in its Phase 3 clinical trial of trigriluzole in spinocerebellar ataxia (SCA)…
There is a must-read for more inspiration about living with sickle cell anemia (SCA) – a young woman’s blog, but not just any blog. She’s a DIVA (as in, absolutely…
As much as it pains me as a “word guy" to admit it, music is probably unparalleled among all the other arts in its ability to motivate action and change…
Want some more news, events and announcements on Sickle Cell? We got 'em! [one_half] [/one_half] [one_half_last] Brazilian Researchers Propose Classification of 3rd Sickle Cell Subtype SAFE Treatment for…
Admittedly, I’m no expert when it comes to sickle cell disease, but after reading about an inspirational young woman who is forging on to live—and I mean LIVE—her life, I’d like…
Need to get your inspiration fix for sickle cell anemia (SCA)? Look no further! A short and simple video is a click away a la the National Institutes of Health…
Bethany Barber is a Virginia native living with sickle cell anemia who has dreams of making it big as a model. She goes on daily castings, as modeling is her main…
Really? Really. As if having one serious chronic disease isn’t enough, what makes God say, “Hmm…Let’s give this poor, innocent child, another chronic illness"?! In this case, I'm talking about…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
