Salarius Pharmaceuticals has just announced the addition of a new clinical trial site for their study investigating seclidemstat as a treatment for Ewing sarcoma (relapsed or refractory) as well as…
Even though some treatments are beneficial for patients, these same treatments can also cause adverse reactions. This seems to be the case for Isturisa (osilodrostat), according to Cushing's Disease News.…
33 year old Emily Helm from Brandon, Mississippi, had always learned that many of her family members on her father's dealt with bone weakness, joint problems, and pain. It was…
Acknowledgment: This story is sponsored by Takeda and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted and valuable information to the…
As we've learned more about individuals facing long-term symptoms from COVID-19, we have heard more and more about how it mimics other chronic conditions. For instance, those suffering from chronic…
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…
From July 2-3 and 8-10, 2021, the European Crohn's and Colitis Organisation (ECCO) held its 2021 virtual Annual Congress (ECCO'21). During the Congress, many discussed new research and trends…
Chronic Fatigue Syndrome Chronic fatigue syndrome (CFS), otherwise known as myalgic encephalomyelitis (ME), is a rare disease primarily characterized by unexplainable and intense fatigue that is not relieved with rest.…
The healthcare system is designed to provide safe, effective, and beneficial care for all. Unfortunately, the current system represents a wealth of socioeconomic and racial disparities. Past studies have shown…
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…
Seven years ago, when Hunter Parrick was 10 years old, he went for what could've been a routine ear surgery. However, during the surgery, doctors found out that Hunter was…
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…
For patients with Cushing syndrome, transsphenoidal surgery can be a beneficial option for addressing pituitary growths or tumors. But according to Cushing's Disease News, this same surgery also increases the…
In the United Kingdom, the Medicines and Healthcare products Regulatory Agency (MHRA) regulates medication, transfusion components, and medical devices, making sure they meet safety and efficacy standards. According to Pharma…
Clinical trials are important research tools to help understand the safety, efficacy, and tolerability of potential treatments. According to a news release from July 19, 2021, biotechnology company Prometheus Biosciences,…
Access to treatment is an issue for patients across the world, especially for those with rare diseases. Laurie Young, who was diagnosed with type 2 spinal muscular atrophy (SMA) at…
About five months ago, Lexi Robins was born in the United Kingdom. She didn't show any signs of abnormal development at first, besides the fact that her thumb didn't move…
According to a recent news release from biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx"), the company's therapy Mepsevii (vestronidase alfa) was approved in Spain for reimbursement. The entire reimbursement process takes…
As reported in Times Union; this was a year where the numbers of many contagious diseases saw steep declines as the world stepped up in handwashing and mask-wearing. In the…
According to the Cancer Network, orelabrutinib, a Bruton tyrosine kinase (BTK) inhibitor, was recently granted Breakthrough Therapy designation within the United States. While the treatment is also indicated for patients…
According to a story from MarketWatch, the biotechnology company Cyclo Therapeutics announced that is has proceeded with the enrollment for its phase 3 clinical trial. This trial will evaluate an…
What do phenylketonuria (PKU), galactosemia, and maple syrup urine disease (MSUD) have in common? All three are considered inborn errors of metabolism, or rare genetic disorders in which the…
In a review posted on dovepress.com, monoclonal antibodies have established themselves as an important tool in the management of multiple sclerosis in both the relapsing-remitting form and the progressive form.…
Ally Winter was diagnosed with short bowel syndrome when she was four years old. Now, she is 17 years old and working to raise awareness for rare and chronic illnesses.…
Happy Friday! This week, we examine the struggle to access experimental rare therapies through compassionate use. Also, in our new Compassion Corner series, we examine why addressing patients' personal wellbeing can…
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