In the past, researchers have questioned whether an association exists between Lyme disease and psychiatric or mood disorders such as depression, anxiety, or even suicidal ideation. While some recognized a…
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…
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…
As of today, the world has been battling the COVID-19 pandemic for over 1.5 years. Throughout the world, the novel coronavirus is responsible for 197 million cases and approximately 4.7…
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…
Despite drug developers' best efforts, the path to approval is not always easy. Ask biotechnology company Ardelyx, whose potential treatment option tenapanor was recently rejected by the FDA. According to…
Dr. Javier Munoz, the director of the Mayo Clinic's Lymphoma program in Phoenix, has recently conducted an interview with Kristie Kahl, the Vice President of content for the Cancer Network.…
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…
Alexis Loveless is a 14-year-old from Louisville, KY who was recently diagnosed with Long QT syndrome. Now, she's doing her best to raise awareness by sharing her own story of…
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…
If I told you that tiny bubbles could be used to more effectively treat cancer, would you believe me? Well, in the case of neuroblastoma, that might be correct. According…
Rare disease patients often face obstacles when it comes to the different aspects of care, whether that's diagnosis, screening, or management. Recent research from Baylor College of Medicine investigated the…
How many of you find yourself rushing around and it’s causing you stress and pain? And if you already have pain, does it add to your pain? Are you trying…
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…
Updates from the Phase 3 clinical trial called FIGHT-302 were just released at the 2021 European Society for Medical Oncology World Congress on Gastrointestinal Cancer. The trial is comparing two…
In the United States alone, an estimated 300,000 people have moderate-to-severe chronic idiopathic urticaria (CIU), or chronic hives which are either recurrent or last for over 6 weeks, which…
Happy Friday! This week, we examine new findings that may reveal why cancer cells are able to resist an attack by immune cells. Also, why is a mother having to…
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…
Previously, biopharmaceutical company XyloCor Therapeutics ("XyloCor") performed the first portion (dose-escalation) of the Phase 1/2 EXACT clinical trial evaluating its investigational treatment XC001 (encoberminogene rezmadenovec) for patients with refractory…
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…
Prior to this year, there were no reported cases of monkeypox in the United States since 2003. This all changed when a Texas man brought the rare disease back with…
According to a relatively recent news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), interim data is now available from a Phase 1 clinical trial evaluating belcesiran for patients with…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
According to a July 27 news release from biopharmaceutical company Day One Biopharmaceuticals ("Day One"), the company's therapeutic option DAY101 received Rare Pediatric Disease designation within the United States. Altogether,…
In a news release from July 21, 2021, biotechnology company Genentech shared that its therapy Venclexta (venetoclax), in conjunction with azacitidine, received Breakthrough Therapy designation from the FDA. Overall, the…
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