Treatment options such as Haegarda and Takhzyro have significantly improved the treatment landscape for people living with hereditary angioedema (HAE). The treatments, used as prophylaxis, work to reduce disease burden…
Roivant Sciences recently shared updates on the Phase 2b TUSCANY-2 study. Within the study, the team was evaluating RVT-3101 for moderate-to-severe ulcerative colitis. RVT-3101 is a fully human monoclonal…
Do mast cells—a type of white blood cell that plays a part in immune and allergic responses—contribute to the development of eosinophilic esophagitis (EoE)? In the past, eosinophils (another…
It is important to improve the treatment landscape for various types of cancer. Currently, there is a wealth of research searching for cancer cures. Until that happens, though, we…
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…
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…
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…
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…
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…
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,…
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,…
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…
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.…
An estimated 2-4% of people worldwide have fibromyalgia, a chronic disorder characterized by musculoskeletal pain, fatigue, and issues with mood, sleep, and memory. This disorder, which affects women more…
Past studies have shown that our gut microbiota, or the collection of microorganisms (bacteria, archaea, fungi, yeast, viruses) within our gut, plays an immense role in our health. Our…
Sometimes it can be difficult to talk about our health. For people living with genital cancers, there are sometimes additional burdens: a fear of discussing a "taboo" topic, concerns…
About 150,000 people with glaucoma across the United States wear contact lenses—and they may soon have access to an even more effective wearable treatment option. According to Medical Device Network,…
In early 2022, the U.S. Food and Drug Administration (FDA) approved Carvykti (ciltacabtagene autoleucel) for the treatment of patients with multiple myeloma. Carvykti is a BCMA-directed T cell immunotherapy.…
If you’ve ever heard of alpha-synuclein before, you may have heard it described in relation to Parkinson’s disease. Alpha-synuclein clumps in the brain; while doctors are still trying to…
As it currently stands, it can be difficult to diagnose a rare disease. Many patients require multiple specialist visits over years (the average rare disease diagnosis takes 4-5 years,…
In late May 2023, clinical-stage biotechnology company AiViva Biopharma Inc. ("AiViva") shared via news release that the company had launched a Phase 1 clinical study evaluating AIV007 for diabetic…
In August 2022, I reported on a story that touched my heart: Lucy Landman's PGAP3 diagnosis. With less than 50 diagnosed cases of PGAP3 worldwide, parents Zach and Geri jumped…
At the very top of the Rensselaer Polytechnic Institute's website reads a bold statement and call to action: "Why not change the world?" As one of the United States' most…
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