Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
The European Medicines Agency (EMA) explains that monepantel is an anthelmintic therapy, or a type of therapy that is used to expel worm-like parasites called helminths or nematodes from the gut.…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Heterozygous familial hypercholesterolemia (HeFH) can be difficult to control on available standard-of-care cholesterol-lowering medications. Therapeutic interventions are needed to reduce high LDL cholesterol levels and improve health and quality-of-life for…
The American College of Rheumatology (ACR) held its annual scientific meeting -- ACR Convergence 2023 -- from November 10 to 15. During the meeting, stakeholders throughout the field shared insights…
Strokes, traumatic brain injuries, amyotrophic lateral sclerosis, Alzheimer's disease, and multiple sclerosis all share something in common: they all have profound effects on the brain and its function. They can…
Loriana has been through more than most. But she hasn’t done it alone. Like so many who have had cancer, Loriana’s battle is a long and complex one. Though she…
The International Congress of Parkinson's Disease and Movement Disorders took place this year from August 27 to 31. During the Congress, researchers and other stakeholders from across the globe…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
In the United States, the FDA grants Fast Track designation to drugs that treat rare and serious conditions or fill unmet needs. The designation helps to facilitate and expedite the…
Having a rare or uncommon condition can be undoubtedly overwhelming. The diagnosis comes with associated mental health, financial, physical, and social burdens that can weigh heavy on your mind. But…
In 2020, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to magrolimab for the treatment of newly diagnosed myelodysplastic syndromes (MDS). This designation is designed to expedite…
English-American guitarist, singer, and songwriter Peter Frampton is best known for his hit album “Frampton Comes Alive!” and his upbeat, immersive performances. But he is also known for his efforts…
According to an article from Akosua Mireku of Pharmaceutical Technology, the U.S. Food and Drug Administration recently granted Orphan Drug designation to SNB-101, a potential therapy for people living with…
You might know Danny Bonaduce for his role as Danny Partridge in "The Partridge Family” or his time as an American radio personality. But Bonaduce can now also call himself…
Orphan Drug designation is granted to therapies that are designed to treat, diagnose, or prevent rare diseases or conditions. In the United States, a rare condition is one affecting fewer…
Raising awareness for rare diseases and conditions is important for many reasons. First, it helps to increase education and understanding around these rare conditions. Awareness also offers support to those…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely…
Although the 5-year survival rate for pancreatic cancer has slightly risen (from 11% to 12%), there is still much work to be done. Pancreatic cancer can be difficult to treat.…
“Three years ago, my lung function was 11% and I was hours from death, then I received the gift of life – which was Trikafta.” These are the words of…
In December 2022, Liam Hendriks—a pitcher for the Chicago White Sox—was diagnosed with non-Hodgkin’s lymphoma (NHL); he shared this news with the world in January 2023, which Patient Worthy reported…
As reported in Yahoo! Finance, the United States’ Food and Drug Administration (FDA) recently granted Orphan Drug designation to MP1032. This therapy, developed by clinical-stage biotech company MetrioPharm, is being…
In 2014, head coach Jimbo Fisher led the Florida State Seminoles to win the BCS National Championship Game. He’s now head coach of the Texas A&M Aggies, recently deemed by…
Pyxis Oncology is driven to improve the lives of people living with cancer through the development of innovative immunotherapy and antibody drug conjugate (ADJ) therapies. Their treatment PYX-201, a novel…
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