43 People in South Africa Have Died of Cholera
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…
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.
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…
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…
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…
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,…
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,…
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…
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…
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…
The American Society of Clinical Oncology (ASCO) held its Annual Meeting from May 31 - June 4, 2023. During the meeting, attendees discussed a variety of oncological topics, trends,…
In January 2023, 23-year-old Angel Anthony Cortez was admitted to the hospital. His health had rapidly deteriorated over the previous days and his family was concerned. Following his admission,…
In May 2023, Pharmaceutical Technology reported that INO-3107, a DNA medicine vaccine candidate developed by biotechnology company Inovio Pharmaceuticals (“Inovio”), received Orphan Drug designation from the European Commission. INO-3107…
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…
When it comes to rare diseases, advancing research is crucial to developing a better understanding of the disease and how it can be treated. However, it can sometimes be difficult…
Zanidatamab is an investigational IgG1 bi-specific monoclonal antibody that targets two non-overlapping HER2 domains. HER2 is overexpressed in a variety of cancer types such as breast cancer and esophageal cancer.…
For as long as he can remember, Eli McCausland has been fascinated by wrestling. His passion for the sport eventually culminated in his co-ownership of Midwest All-Star Wrestling, as well…
TransCode Therapeutics, Inc. (“TransCode”) was founded with a mission to revolutionize the cancer treatment landscape and improve patient outcomes. The company is currently working to uphold this mission through the…
When it comes to the rare disease landscape, there is not enough conversation discussing mental health needs. People undoubtedly need more mental health support and resources during the diagnostic odyssey.…