As reported in Michael J. Fox News, the FDA has just approved the novel Percept PC Neurostimulator, a device inserted into the brain of patients with Parkinson's to administer deep…
After forty years of research to counter or cure acute myeloid leukemia and other blood cancers, a few years ago the medical community exploded with eight new cancer drugs.…
In a recent press release, the U.S. Food and Drug Administration (FDA) announced their approval of Inqovi for patients with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). The…
BioMed Central recently featured a pilot study conducted by a team at Japan’s Hitachi General Hospital department of critical care and emergency medicine. Their pilot study is based on…
A team of researchers from the University of Alberta have created a possible treatment for facioscapulohumeral muscular dystrophy (FSHD). Their work has been published in the Proceedings of the National Academy…
July is recognized as Juvenile Idiopathic Arthritis Awareness Month. Though arthritis is often considered an illness that is associated with old age, it is estimated that around 300,000 children in…
Launched in January of 2020, and ending in May, the BEAT-PD Dream Challenge was designed to understand whether passive data collected from sensors could track symptom severity and disease progression…
Timber Pharmaceuticals focuses on rare diseases of dermatologic origin. These include scleroderma, congenital ichthyosis, tuberous sclerosis complex, and facial angiofibromas. They are currently working on two clinical trials. Trial 1…
CMT1A Charcot-Marie-Tooth disease (CMT) has a few different subtypes. 60% of all patients diagnosed with type 1 of the rare disease have CMT1A. This subtype means patients have an duplicate copy…
As you probably know, our bodies are extremely complex. Every day, there are multiple processes working to help us live, breathe, work, eat, and more. But did you know…
Throughout 2020, the medical community and much medical research has been centered around COVID-19, a novel coronavirus turned global pandemic. With just around 11 million diagnosed cases, COVID-19 presents…
Parkinson’s News Today recently published an article containing new information about biomarkers in Alzheimer’s disease that can be linked to the early stages of Parkinson’s disease. Many people with…
Each and every day, the medical community (and the world) learn more about COVID-19. What began as a novel coronavirus in Wuhan, China has turned into a global pandemic…
According to a recent press release, EspeRare received Breakthrough Therapy Designation for its investigational therapy ER-004, designed for the prenatal treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED). Ultimately, this…
In some cases, people have a medical condition that doesn't respond well to treatments. The journey to finding an effective treatment can be long and difficult. But for patients…
According to ASH Clinical News, a phase 1 trial of lenzilumab illustrated clinical benefits for approximately one third of participants with chronic myelomonocytic leukemia. It is a novel antibody, and…
When it comes to researching and finding treatments for rare diseases, funding plays a crucial role. Recently, the Medical Research Future Fund (MRFF) Stem Cell Therapies Mission presented Monash…
There are multiple forms of Batten disease, each with their own distinct genetic cause. One of these forms is caused by a mutated CNL6 gene, but researchers were unsure as…
One huge goal throughout the medical world is to find effective and well-tolerated treatments for patients with cancer. According to News Medical, researchers recently discovered one beneficial treatment for…
According to a story from The Boston Herald, a new treatment for acromegaly called Mycapssa was recently approved by the US Food and Drug Adminstration (FDA). Developed by Chiasma, a…
Since the dawn of time, the human body has changed to cope with microbes and environmental changes. As a result, specific gene mutations evolved to protect populations from harm.…
In 2019, Alnylam Pharmaceuticals received FDA approval for Givlaari, the first RNAi therapy for patients with acute hepatic porphyria. Although some were initially worried about the therapy's safety and…
According to a story from BioSpace, the drug company Zynerba Pharmaceuticals Inc. recently released the results from a clinical trial. This trial was testing the company's product Zygel™ (CBD gel)…
David Fajgenbaum toes the line between patient and doctor. After growing up with a physician as a father, Fajgenbaum chose to follow in his footsteps. His most important lessons came…
In 2016, 6-year-old Alec Ahmad loved club soccer, playing games with his two brothers, and running around with his friends at recess. He was full of energy. But every once…
© Copyright 2024 Patient Worthy
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.
