Fulcrum Therapeutics recently announced the interim results from their Phase 1 trial of FTX-6058 in a press release. This selective small-molecule inhibitor of EED is in development for the treatment…
The FDA has recently granted ONCOFID®-P the Orphan Drug designation for the treatment of malignant mesothelioma (MM). This designation is reserved for therapies indicated for rare diseases, and it provides…
HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma patients haven't seen a drug approval for their disease state in a decade. That is until the FDA approved Keytruda in combination with…
Patients with relapsed or refractory mantle cell lymphoma (R/R MCL) may have a new treatment option, based on positive results from a Phase 1/2 trial of Zanubrutinib published in Blood…
A local radio host and police dispatcher known as Dr. Rock to his audience in Bakersfield, California has recently passed away due to multiple system atrophy (MSA). You may recognize…
Recently, the FDA approved an investigational new drug (IND) application for '1104, a first-in-class peptide for the treatment of eosinophilic esophagitis (EoE). Now, Revolo Biotherapeutics will move forward to investigate…
The FDA has recently granted Orphan Drug designation to TST1001, Transcenta Holding Limited's monoclonal antibody for gastric cancer/gastroesophageal junction (GC/GEJ). Prior research has demonstrated TST1001's anti-tumor activity, and ongoing research…
Renal cell carcinoma (RCC) is typically treated with single-agent tyrosine kinase inhibitors (TKIs). Now, this may be changing as research demonstrates the potential of the combination of TKIs and immunotherapy.…
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…
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…
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…
Having a rare, chronic disease can impact one's life significantly, whether it's physically, spiritually, socially, or psychologically. Not only this, but those responsible for these patients' care are affected as…
The European Medicines Agency (EMA) recently received a Marketing Authorization Application (MAA) for Mycapssa, a maintenance therapy for acromegaly. Chiasma submitted the application, basing it on the results of their…
A better comprehension of one's cancer can provide improved treatment outcomes. It's easier to fight something that one understands. That's why a recent article published in Medical Xpress is so…
Underserved communities have difficulties when trying to access the resources that are available to the majority. Because of this, more work has to be done to ensure that everyone can…
The FDA has recently approved KEYTRUDA (pembrolizumab) in combination with LENVIMA (lenvatinib) for the treatment of advanced endometrial carcinoma that is not mismatch repair deficiency or microsatellite instability-high. This approval…
The FDA has recently granted their approval to a Phase 2 trial of FHL-301, a treatment for those in the earlier stages of Parkinson's disease. Forest Hills Lab will conduct…
The European Commission recently approved of Enspryng for neuromyelitis optica spectrum disorder (NMOSD). Now, European patients have a new treatment option. This one is especially useful as it can be…
About a month ago, from June 23-26, The Immune Deficiency Foundation hosted the 2021 Primary Immunodeficiency Conference. The event was the largest gathering of primary immunodeficiency (PI) patients across the…
OncLive recently interviewed Daniel Pollyea, MD, MS, about the current treatment landscape for higher-risk myelodysplastic syndromes (HR-MDS), along with exciting new data from a Phase 2 trial of pevonedistat plus…
If you have ever donated to the American Red Cross, you've probably been receiving a rash of calls and emails asking for donations. I know I have. This is because…
Community support can be extremely uplifting and important, especially when one is impacted by a rare disease. This is the case for three-year-old Charlie Fry, who was born with SLC6a1.…
It's the season of sun and summer fun, which leads many to enjoy the great outdoors and warm weather. While this is all in good fun, it's important to remember…
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…
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