The month of April is recognized as Sarcoidosis Awareness Month, a time for spreading awareness about sarcoidosis among the general public and the medical community. The Foundation for Sarcoidosis (FSR)…
Recently, the Cancer Foundation of Santa Barbara (CFSB) was given a $500,000 grant courtesy of the Dr. Howard R. Bierman and Anthony Granatelli Fund. Altogether, the fund is designed to…
According to Yahoo Finance, immuno-oncology company Surface Oncology ("Surface") recently received Orphan Drug designation for its leading therapeutic candidate SRF617. Altogether, this therapy is designed to treat patients with pancreatic…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Orphazyme's latest trial has ended in failure after arimoclomol, a possible treatment of inclusion body myositis (IBM), failed to meet any of its endpoints. As the drug has shown massive…
In a recent press release, clinical stage biotech and biopharmaceutical company Biom Therapeutics ("Biom") shared that its drug candidate BIO017 was granted Orphan Drug designation. The treatment, designed for patients…
At the very end of March 2021, specialty pharmaceuticals company JCR Pharmaceuticals Co., Ltd. ("JCR") shared that its investigational drug candidate JR-171 received Orphan Drug designation from the European Medicines…
Cancer Network recently reported that Jazz Pharmaceuticals’ Vyxeos was approved by the FDA to treat therapy-related pediatric patients one year or older who were newly diagnosed with acute myeloid…
As Parkinson’s diseases slowly erodes a person’s control of their movement, many normal parts of life become much more difficult. In a new study in Brazil, “Evidence-Based Recommendations for…
A reader named Fiorella, recently reached out in response to an article we wrote entitled: A Common Parasite With An Uncommon Name: Toxoplasma Gondii is Associated with Brain Cancer. This…
Researchers from the University of Barcelona have recently found a new biomarker which may be able to diagnose Parkinson's disease sooner. This biomarker is a protein fragment called GPR37. It…
Sometimes it's hard to pick apart the cause of symptoms. Am I anxious because the bus is late or because Covid-19's insidious shadow is still lingering all these months later?…
Around 30% of all patients with relapsing-remitting multiple sclerosis (RRMS) experience some type of cognitive impairment or decline at the time of diagnosis. Around 50% of patients experience some of…
According to Yahoo Finance, D&D Pharmatech has initiated dosing in their Phase 1 trial of DD01, a treatment for nonalcoholic fatty liver disease (NAFLD) and diabetes in obese patients. As…
It takes courage to suspect that there is something wrong and doctors are misdiagnosing your symptoms. It takes courage to keep going back and politely but firmly asks for more…
Spinal muscular atrophy (SMA) patients aged two months and older will now have an at-home treatment option in Europe. The European Commission (EC) has approved of Evrysdi for those who…
Primary Sclerosing Cholangitis Primary sclerosing cholangitis (PSC) is a rare condition which causes inflammation within the bile ducts of the liver. It can lead to cirrhosis of the liver or…
Currently, there is no cure for Crohn's disease, and treatment options, like surgery, can sometimes be invasive. Additionally, treatments like anti-inflammatory drugs or nutritional supplements treat the symptoms, but not…
Financial Burdens in Rare Disease In 2019, 20% of all adults in the United States have reported that they had large medical bills that were unexpected. 18% currently had medical…
By May 25th, 2021 the FDA will announce their decision regarding approval of Opdivo used in combination with chemotherapy for gastrointestinal cancers. Back in December, Priority Review was granted to…
Aeglea Biotherapeutics has just announced a new collaboration with Immedica to bring pegzilarginase to other countries outside of the United States. They have created a license and supply agreement to…
In a study published on Hindawi, a team of scientists have discovered that the C-reactive protein to albumin (CAR) ratio can be used to predict the risk of complications following…
Lyme disease, and other tick-borne illnesses, can cause intense and prolonged symptoms. In fact, some patients experience chronic symptoms for a number of years following the initial bite. However,…
Recently, the National Cancer Institute provided a $2.5M grant to support specialized medical research being performed at the Wake Forest Organoid Research Center (WFORCE). According to the press release,…
Sometimes it can be difficult to find or afford certain medications designed for your condition. However, this may soon be changing for Canadians with spinal muscular atrophy (SMA). On March…
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