Biotechnology company Talaris Therapeutics, Inc. submitted an Investigational New Drug (IND) application to the FDA for their novel cell therapy treatment FCR001. As per a press release, the FDA…
Rare pediatric disease designation has just been granted to Rhythm Pharmaceuticals for their investigative therapy called setmelanotide. This drug is being investigated as a therapeutic option for two conditions- leptin…
In a July 9 press release, the Familial Chylomicronemia Syndrome (FCS) Foundation announced its support for a bill introduced by Congressman Paul Tonko (D-NY) and David B. McKinley (R-WV).…
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…
Each year, June 13th is celebrated as Gastrointestinal Stromal Tumors (GIST) Awareness Day. The ultimate goal of awareness days for rare diseases is to help inform both the general public…
As COVID-19 grips the world, scientists are chasing after it to find the full spectrum of effects as it interacts with human biochemistry. While known for its characteristic cough and…
Alaina Johns, a Philadelphia based freelance writer, wears two hats. One hat as editor-in-chief of the Broad Street Review, and the other as a patient with several chronic illnesses.…
Patients with inoperable or metastasized neuroendocrine tumors (NETs) may now benefit from recently FDA approved peptide receptor radionuclide therapy (PRRT) according to a recent article in MedPage Today. 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…
Happy Friday! The summer heat is setting in, so why not stay in where it's cool and read the latest rare disease news. First, we want to bring attention to…
For many people with Parkinson’s disease, “levodopa” is well-known. Parkinson’s disease is a central nervous system disorder caused by the death of dopaminergic neurons. As a precursor to dopamine,…
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…
At one point in his journey with cystic fibrosis, Marc Cotterill experienced a rapid loss in lung function. In fact, it dropped to just 29%. As his health worsened,…
Medtronic’s Percept is the fourth deep brain stimulation (DBS) system that has been approved by the FDA according to a recent article in Parkinson’s News Today. The unique feature…
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…
According to a story from thv11.com, Susan Head of Little Rock, Arkansas is a marine veteran who now faces a new battle: Castleman disease. In the midst of the ongoing…
Around 10 million people worldwide are living with Parkinson's disease, a progressive nervous system disorder impacting movement. While there are many current and proposed treatments, the medical community continues…
After three years, patients with Leber hereditary optic neuropathy (LHON) who had been treated with LUMEVOQ experienced sustained safety and efficacy. According to a press release from GenSight Biologics,…
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…
Well-known characteristics of aging are the loss of skeletal muscle mass and a general decline in all of the organs in the body. The speed of decline coincides with the…
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…
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