The month of August is recognized as Psoriasis Awareness Month, a time of year set aside to help spread awareness about the disease among the general public and in the…
To the vaccinated, it may feel like the COVID-19 pandemic is finally over, but a convergence of the Delta variant, rising long COVID cases, a higher number of breakthrough…
FoundationOne CDx is a comprehensive genomic profiling test that is tissue based. It is used to identify individuals diagnosed with ALK+ non-small-cell lung cancer (NSCLC) who will be eligible for…
The month of August has been recognized as Autoinflammatory Awareness Month since 2015. This event is centered on spreading awareness about autoinflammatory illnesses among the medical community and the general…
Huafrid Billimoria is a 25-year-old triathlete in India. He has just become the first person diagnosed with cervical dystonia in the country to complete a 42km marathon. In 2019 he…
A recent study has examined what risk factors exist for patients diagnosed with myotonic dystrophy, the most common of all muscular dystrophies. This is a progressive, hereditary condition. Regarding cardiac…
A Phase 3 clinical trial for Non-Germinal Center Diffuse Large B-Cell Lymphoma in patients where it was previously untreated have just announced they are still recruiting participants. The trial is…
According to a story from express.co.uk, multiple sclerosis (MS) is a neurological illness that can have major impacts on the lives of its patients. As a condition that can be…
On July 28, 2021, biopharmaceutical company Thetis Pharmaceuticals ("Thetis") shared that its small molecule Resolvin E1 (RvE1) therapy, TP-317, received Orphan Drug designation from the FDA. While TP-317 has…
Orphan Drug designation is a status granted by the FDA to drugs or biologics intended to treat patients with rare conditions. In this case, the FDA defines "rare" as…
According to a story from Market Watch, the biopharmaceutical company Ultragenyx Pharmaceutical, Inc. recently announced that it was given Orphan Drug designation from both the US Food and Drug Administration…
Sometimes, clinical trials do not go the exact way that is expected. According to Targeted Oncology, this is what happened in a Phase 1b clinical trial evaluating RVU120 for…
Bacterial meningitis is a severe condition that is linked to high mortality. Unfortunately, newborns are much more susceptible to bacterial meningitis. In fact, this infection occurs more than 30 times…
According to a story from MSN, mother Vanessa Ruiz is facing a terrifying choice: feed her family or pay for the genetic testing to confirm that her children Faith and…
According to a press release published on Globe Newswire, the biopharmaceutical company Saniona has announced recently that its investigational therapy Tesomet has recently earned Orphan Drug designation from the US…
Following several Patient Worthy articles on the subject earlier this year, Fierce Biotech recently published news that Biogen is preparing to release the Phase III VALOR trial drug, tofersen,…
According to a story from USA Today, Mark Hoppus, bass player for the iconic pop-punk band Blink-182, was recently diagnosed with a rare cancer: diffuse large B-cell lymphoma. His cancer…
Researchers from Boston University and Boston Medical Center have recently uncovered that base editing is able to correct the gene mutation which causes alpha-1 antitrypsin deficiency (A1AD). This condition impacts…
A study conducted by researchers at Nagoya University in Japan and researchers from Yale University in the United States was recently published in the European Respiratory Journal. This study utilized single-cell…
Amyloidosis is a rare disease which can cause organ failure. It is the result of an abnormal buildup of amyloid proteins in the blood. These proteins are then deposited within…
Dr. Jerry Vockley from the UPMC Children's Hospital of Pittsburgh has been studying the rare disease VLCAD for years. His work has led to the creation of two therapies which…
Many individuals in our day and age find it difficult to sleep. Although the first humans awoke with the sun and slept with the moon, we don't operate that way…
A new clinical trial is starting for Friedreich's ataxia (FA). This trial will investigate if supplementation of vitamin D in its active form, also known as calcitriol, will aid in…
The International Pemphigus & Pemphigoid Foundation (IPPF) has recently announced that is has partnered with the American Dental Association (ADA) for a continuing education course that will help dental professionals…
July 13th 2021 will be recognized as GIST Awareness Day. This is a day set aside to help spread awareness about gastrointestinal stromal tumor (GIST), a rare disease, among the…
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