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…
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.…
KRYPOLIS (carfilzomib) recently received conditional approval in China for the treatment of relapsed/refractory (R/R) multiple myeloma. Licensed by BeiGene, this drug is indicated for adults with R/R multiple myeloma who…
Medical professionals are still working to uncover the cause of Alzheimer's disease. While they believe that a combination of genetic and environmental factors paired with certain lifestyle choices leads to…
Accessing treatments can be difficult, especially if one is impacted by a rare disease. In fact, only 5% of rare diseases have an FDA-approved treatment. Because of this, many patients…
According to MedPage Today, the FDA sent a letter to the manufacturer of the Johnson & Johnson COVID-19 vaccine on Monday containing a warning about its link to Guillain-Barre syndrome…
The CDC is currently looking into three cases of melioidosis throughout the United States, according to an article published in MSN. This rare, bacterial infection is mainly found in the…
We know that it can be difficult to receive the correct diagnosis for a rare disease. Sometimes, you have to be your own advocate. This means learning everything you can,…
Amyotrophic lateral sclerosis (ALS) is a rare, neurological disorder that is characterized by the progressive death of nerve cells and muscle weakness. As time passes, many affected individuals experience difficulty…
Rare diseases face many obstacles when it comes to research and drug development. Small patient populations, a lack of awareness, and limited funding are just a few examples. When it…
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