NXP800 for Cholangiocarcinoma Earns Orphan Drug Designation
In the United States, the FDA grants Orphan Drug designation to drugs or biologics that are intended to treat, diagnose, or prevent rare conditions. A rare condition is one…
Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
In the United States, the FDA grants Orphan Drug designation to drugs or biologics that are intended to treat, diagnose, or prevent rare conditions. A rare condition is one…
Retinoblastoma is a rare cancer that begins in the retina, or the sensitive lining in the back of your eye. While it can affect people of all ages, it is…
245 words (source - 6% match) vs. 386 words (mine - 4% match) In the European Union, Orphan designation is granted to therapies intended to treat or use for a…
The 2023 Annual Meeting of the Myotonic Dystrophy Foundation took place from September 7-9 this year. During the meeting, stakeholders gathered in the nation's capital to discuss research, trends,…
As many people within the rare disease community know, a rare disease diagnosis can come with a significant financial burden. Additionally, the costs, treatments, equipment, and other needs are not…
Previously approved for the treatment of narcolepsy, pitolisant (marketed under the brand name Wakix) may soon be leveraged for another rare condition: idiopathic hypersomnia (IH). Pitolisant is a histamine 3…
INT230-6, developed by clinical-stage biotechnology company Intensity Therapeutics (“Intensity”), is an investigational therapy comprising three moieties: cisplatin, SHAO-FA (a penetration enhancing molecule), and vinblastine sulfate. A moiety, in this sense,…
Also known as Richter’s Transformation, Richter syndrome is a rare and aggressive complication of chronic lymphocytic leukemia (CLL). When someone develops Richter syndrome, their CLL can transform into another rare…
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…
Teegan is Kelly and Matt Olson’s youngest daughter, so when she began missing developmental milestones, her parents were on alert. After a variety of tests over the span of…
Currently available therapeutics may not be adequately effective in the fight to treat late-stage acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). Researchers and drug developers are working to identify…
Galila Yohannes’ parents, who originally hail from Eritrea, brought their family to Israel for a better life. And while they never would have expected what came next, this move likely…
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 U.S. FDA has approved two treatments for people living with idiopathic pulmonary fibrosis (IPF). These therapies—Esbriet (pirfenidone) and Ofev (nintedanib)—both work to reduce fibrosis, or scarring, in the…
In Jordyn Sava's reporting from Targeted Oncology, readers learn that the U.S. Food and Drug Administration (FDA) granted clearance to a global Phase 3 study. This means that the…
Unfortunately, gaining access to care within the rare disease space can be difficult. There is often lesser education and awareness about rare conditions, less research performed, and poorer access to…
21-year-old Chloe Feighery has a deep love and appreciation for her younger sister Heidi, who is just one and a half years old. When she learned that she was becoming…
Soon after Emma Young was born, she was diagnosed with a rare genetic disorder called Bannayan-Riley-Ruvalcaba syndrome (BRRS). At two months old, she battled COVID-19. Then she was diagnosed with…
For the first year of his life, Warner Kays looked like an average, healthy, happy baby. He crawled around, took joy in family time with his older sister Presley, and…
Have you ever heard of CD55-deficient protein-losing enteropathy? This rare condition, which is also called CHAPLE disease, was only identified in the past 10 years. Right now, there are only…
When Crystal York learned that her daughter Harmony had a rare genetic disease called DHX30, she was shocked. Even more shocking is that Harmony is just one of 40…
Chorea, a characteristic manifestation of Huntington's disease, refers to uncontrolled "dance-like" movements in the fingers, feet, face, or torso. These movements may worsen in times of heightened emotion or…
At the moment, there are limited treatment options for glioblastoma, a rare form of brain cancer. While surgery, radiation, chemotherapy, and electric-field therapy are all utilized in the fight…
The Phase 2 EXHALE-1 clinical trial, initiated by clinical-stage biotechnology company Areteia Therapeutics, Inc. ("Areteia"), sought to understand how safe, effective, and well-tolerated dexpramipexole was in reducing symptoms of…