ChemoCentryx has just announced that they have submitted their New Drug Application to the FDA for a novel ANCA-associated vasculitis therapy. It is called avacopan and it's finally something non-steroidal.…
According to a study on biorxiv.org, a method called intranasal mechanical stimulation (INMES) appeared effective in reducing symptoms associated with myalgic encephalomyelitis (also called chronic fatigue syndrome) by around 30…
By Lauren Thayer from In The Cloud Copy Nonalcoholic fatty liver disease or NAFLD is a term used to describe a range of conditions in which a patient’s liver has…
By Danielle Bradshaw from In The Cloud Copy The Lancet Psychiatry has published a review stating that although there have been signs demonstrating that delirium may be a common symptom…
According to an article in Globe NewsWire, Onconova Therapeutics, a biopharmaceutical company currently focusing on myelodysplastic syndromes, recently announced results of its Phase 1 study of oral rigosertib in…
By Lauren Thayer from In The Cloud Copy Tourette syndrome (TS) is a neurological disorder that is characterized by involuntary, repetitive movements and vocalizations. These movements and vocalizations are called…
By Lauren Taylor from In The Cloud Copy Biogen announced new data from the NURTURE trial of pre-symptomatic patients with spinal muscular atrophy (SMA). SMA is a rare, autosomal recessive…
Within the medical realm, clinical trials play a crucial role in understanding disease progression, medication safety and efficacy, and patient outcomes. Recently, PTC Therapeutics began a Phase 1 clinical…
According to Parkinson's News Today, the Parkinson's Foundation has released data from the PD GENEration study that shows that genetic testing is feasible for the larger Parkinson's community. As around…
The FDA has recently approved pembrolizumab, which is a treatment for colorectal cancer, specifically MSI-H and dMMR colorectal cancer. This approval comes after the release of positive results of the…
According to a story from GlobeNewswire, the biopharmaceutical company Onconova Therapeutics, Inc. has recently published results from a phase 1 clinical trial that was sponsored by the company. This trial…
Data has recently been published to support a new diagnostic method for progressive supranuclear palsy, according to GlobeNewswire. Life Molecular Imaging and AC Immune SA have reported this data, which…
According to a story from Medscape, a recent cohort study published in the journal Cancer has concluded that updates are needed to the staging classification currently in use for renal cell carcinoma…
By Jodee Redmond from In The Cloud Copy When a patient identified as Ms. B in a case history complained of headaches starting at the back of her head and…
Diagnosing Parkinson's disease can be difficult, as it can only be done after motor symptoms appear. In an effort to create a better diagnostic method that can catch the disease…
According to a recent article in EurekaAlert, researchers at the University of Boston recently published their new study investigating ROBO2. The ROBO2 signaling pathway has emerged as a therapeutic…
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…
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…
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…
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…
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…
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)…
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.
