Right now, there are no available therapies for people living with Fragile X syndrome (FXS). However, clinical-stage biotechnology company Connecta Therapeutics (“Connecta”) is working to change that. In a recent…
“I never thought that it could happen to me.” Unfortunately, we tend to repeat this refrain when facing unexpected hardships in our life from trauma to illness. For many people,…
Each year, an estimated 21,000 to 143,000 people around the globe die from cholera. This acute diarrheal illness, caused by Vibrio cholerae (V. cholerae) infection, is more common in…
BCMA is a protein that is found in multiple myeloma cancer cells. CAR-T cell therapies such as Carvytki are engineered to home in on BCMA proteins. According to a…
Your HTT gene encodes for the production of huntingtin, a protein that helps your neurons (nerve cells in your brain) function correctly. When mutations happen in this gene, the protein becomes…
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…
Clinical trials are incredibly meaningful in advancing research, understanding, and potential treatments for many conditions. Unfortunately, these trials are not always successful, meaning that the evaluated drugs cannot be considered…
uniQure, a leading gene therapy company, recently shared an update on a Phase 1/2 clinical study evaluating AMT-130 for Huntington’s disease. The safety and proof-of-concept study enrolled 26 participants with…
When someone is diagnosed with a rare disease, cancer, or really any health-related issues, they tend to question what comes next. How will this condition impact them? What is…
As of 2020, there were about 241 million cases of malaria occurring on a global scale with a death toll of 627,000. These cases are most common in areas…
According to a recent article in the Pharma Times, E02463 is a therapy for indolent or slow growing, not yet problematic non-Hodgkin B cell lymphoma (iNHL). Enterome, its developer,…
Medical research has yielded a significant number of breakthroughs that have advanced the field and helped people in need. But in the rare disease sphere, it has sometimes been…
Rheumatoid arthritis, lupus, multiple sclerosis, and myasthenia gravis are all considered autoimmune disorders. These conditions occur when the immune system, which normally protects the body against infections or other…
As the world becomes increasingly technological, researchers and scientists are leveraging these advances for scientific improvement. Artificial intelligence (AI) is now being used to develop products, screen drug candidates,…
According to a story from statnews.com, a new documentary film from Elle Brooks-Tao titled "I'm Not Crazy, I'm Sick" delves into the stories of three people living with long-term Lyme…
The European Congress of Rheumatology (EULAR) took place from May 31 - June 3, 2023. During the Congress, experts in rheumatology came together to discuss research, new guidelines, and…
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…
There are current approved therapies for people living with wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME). For example, doctors may prescribe VABYSMO, Lucentis, Beovu, Avastin,…
According to a recent article in People Magazine, this Illinois family has a dream: to turn amyotrophic lateral sclerosis (ALS) from a fatal to a chronic illness in Brian’s…
Nobody expects to hear that their child, especially their baby, has cancer. But that is just what Chris and Carney Miller were faced with in 2023. The Miller family…
According to a story from BioPharma Dive, Apellis Pharmaceuticals will now join the ranks of pharma companies that have seen an investigational therapy being developed for amyotrophic lateral sclerosis falter…
Throughout the world, it can be difficult to advance research and drug development in the rare disease space. In part, this is because many rare diseases have small population…
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…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
It can be undoubtedly difficult to identify therapies for rare diseases. Between the cost of research, the small population sizes, and the time required, the drug development process can…
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