At Japan’s Fujita University, researchers reported to Inside Precision Medicine that a new risk measurement may add a layer of accuracy in predicting sudden cardiac death. The measure, called estimated…
As I reflect on over 20 years living with dystonia and pain and other symptoms that changed my life in many ways, I want to share some of the things…
In some cases, dermatomyositis can be difficult to treat. A variety of treatment options are available, from corticosteroids and immunosuppressants to antimalarials, colchicine, alendronate, and warfarin. But this rare condition…
Have you ever heard of cytokines? These signaling proteins play a role in inflammation in your body. Normally, cytokines help your immune system respond to foreign invaders. But when too many…
By: Harsha K Rajasimha, Ph.D., Founder and CEO of Jeeva Clinical Trials Challenges Unique to Cell and Gene Therapies (CGTx) Clinical Trials Cell and gene therapies (CGTx) have emerged as…
The month of August is recognized as Spinal Muscular Atrophy (SMA) Awareness Month. This is a time of year in which the community works to spread awareness about this rare…
Rare disease drug development can be challenging. Countries globally developed programs to incentivize innovation and development in this space. One of these initiatives is the designation of orphan drugs, or…
On July 11th Dr. Mathew Winton went to Capitol Hill with senators and staff from both parties to discuss collaboration with the FDA regarding the pending termination of the Priority…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with professional speaker and…
The long-awaited positive results of Annexon Inc.’s Phase 3 trial investigating ANX005 for Guillain-Barré syndrome (GBS) was presented at the PNS 2024 Annual Meeting. Several leading global GBS experts spoke…
Ruby Carr has always loved playing outside. But an enjoyable day in the summer sun turned into chaos after Ruby was unknowingly bitten by a tick. As the tick latched…
In unedited, early-access research published in Nature, a team of researchers explain that an estimated 60% of people with neurodevelopmental disorders remain undiagnosed, despite comprehensive genetic testing options. In many cases, this is…
Yusara Ahmed was prepared to take action when she learned that her son Yusuf was diagnosed with beta thalassemia, an uncommon blood disorder. Yusara's sister also lived with the disease,…
Recently there has been an increasing amount of research attempting to control CRISPR gene-editing systems. These systems are found in many types of bacteria as a defense against viruses. The…
Clinical-stage biopharmaceutical company Savara Inc. was founded on a desire to develop innovative therapies for people living with rare respiratory diseases. The company's work over the past few years centers…
According to a story from PR Newswire, the biotech company Mission Therapeutics was just awarded $5.2 million in funding to support MTX325, the company's investigational Parkinson's disease therapy. Intended to…
The COVID-19 pandemic took our world by storm. COVID-19 affected hundreds of millions of people globally and was associated with tens of millions of deaths. Today, people are still dying…
According to an article on Pharma Biz, Johnson & Johnson recently submitted a supplemental Biologics License Application to the US Food and Drug Administration (FDA). This application was for the…
Shanghai's Junshi Biosciences Co. (The Company) recently announced in Biospace, that its product, toripalimab (TUOYI®), combined with bevacizumab as a first-line therapy for hepatocellular carcinoma (HCC), met its endpoints in a…
According to an article in Science Daily, a drug currently approved to treat the common skin condition eczema may help provide symptom relief in more serious skin diseases as well.…
Ocugen, Inc., a biotechnology company based in Malvern, Pa. recently reported to BioSpace that DSMB gave a favorable review of its Phase ½ ArMaDa clinical study of OCU410. Approximately 10…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Zika virus, a mosquito-born illness, is not harmful in many cases; for most people, Zika virus causes either no symptoms or a mild sickness. However, this viral illness is extremely…
The American Academy of Sleep Medicine and the Sleep Research Society held the 38th annual SLEEP meeting in early June 2024. The meeting allows stakeholders across the sleep sphere to…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
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