Jazz Pharmaceuticals plc has taken a major leap in rare cancer treatment with the recent FDA accelerated approval of Modeyso (dordaviprone), the first-ever therapy for relapsed H3 K27M-mutated diffuse midline…
August 2025 was a landmark month for rare disease innovation, as the U.S. Food and Drug Administration (FDA) granted four historic approvals, each representing a first for their respective conditions,…
Right now, there is an urgent need to identify and develop treatments for people with malignant gliomas, including glioblastoma. Unfortunately, there are limited effective treatment options; these cancers are…
At ASCO’s 2023 convention for patients diagnosed with glioma and a specific genetic alteration, Servier Pharmaceuticals announced that vorasidenib, the drug being investigated in its Phase III trial (INDIGO)…
It’s no secret that, in many cases, funding for rare disease research and drug development is slim; the financial burden often falls to families and patients. 40 years ago, the…
Diffuse midline glioma (DMG) refers to a rare and extremely aggressive brain tumor that is most common in children. Chemotherapy and radiation are the current standards-of-care. Unfortunately, even with…
According to a September 2022 article from OncLive, the European Medicines Agency (EMA) granted Orphan Drug designation to CAN-2409. This therapy is being developed as a potential therapeutic option for…
Have you ever heard of Orphan Drug designation? In the United States, this designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics intended…
Currently, no approved treatment options exist for children with pediatric low-grade glioma, the most common brain tumor found in children. However, shares Day One Biopharmaceuticals ("Day One") in a news…
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,…
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…
Did you know that a common food-borne illness might increase the risk of developing a type of rare brain cancer called glioma? According to Science Daily, a recent study suggests…
Monitoring tumors is an essential part of cancer treatment; it allows doctors to make informed decisions regarding which therapies to use. For many forms of cancer, medical professionals have been…
When Lily Wythe, a 14-year-old from Eastwood in England, was diagnosed with diffuse pontine glioma (also known as DIPG), her friends and family knew they had to do something to…
The European Organization for Research and Treatment of Cancer (EORTC) has been evaluating zotiraciclib, which is a new treatment for glioma. This drug has recently been granted the Orphan…
Nobody expects a shocking diagnosis for their significant other right before one of the most important days of their lives. Ariona Kryeziu's life was altered significantly when her fiancee, 20-year-old…
According to a press release from Mateon Therapeutics, Mateon's recent merger agreement with PointR Data establishes plans to combine the use of artificial intelligence and data collection for the development…
Glioma is a type of cancerous tumor that originates in the brain. It has various types and the tumors range in aggressiveness. There are around 11,000 people just in the…
According to Medical Xpress, Chris Jones from the Institute of Cancer Research in the UK has delved into the complexities of the rare childhood brain tumors called diffuse intrinsic pontine…
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