The EMA has recently published their recommendation for extending the indication of a treatment called Nucala to include patients diagnosed with relapsing-remitting or refractory eosinophilic granulomatosis with polyangiitis (EGPA). Specifically,…
NOTA, The Network of Tyrosinemia Advocates, held their 4th Annual NOTA Experience, a family conference in September, despite the extraordinary constraints of time, a pandemic, transportation challenges, and the special…
The FDA has recently approved Skytrofa as a treatment for children at least one year of age who are facing growth failure as a result of an inadequate secretion of…
The FDA has recently approved a new diagnostic tool to help determine which IDH1-mutated cholangiocarcinoma patients may be a good fit for the novel IDH1 inhibitor called ivosidenib. The new…
A recent study has investigated the association between cardiovascular disease risk factors and fat distribution. Researchers used linear regression analyses and adjusted for insulin sensitivity and other factors. Previous research…
My daughter, Carson, was born in June 2020. I received a call 1.5 weeks later about Carson’s abnormal newborn screening. I was told it was most likely an error, but…
September is Charcot-Marie-Tooth disease (CMT) Awareness Month! The Charcot-Marie-Tooth Disease Association has put together 5 different ways you can help spread awareness of this rare disease this month. The Association…
Rare Disease Week this year was July 14th-22nd and held virtually. With over 600 participates advocating for those living with a rare disease diagnosis and 250 patient organizations, this event’s…
CAR T-cell therapy has been used as a treatment for a variety of rare diseases including relapsed or refractory diffuse large B-cell lymphoma (DLBCL), non-Hodgkin lymphoma (NHL), as well as…
Results from a recent Phase 3 trial for diffuse large B-cell lymphoma (DLBCL) have just been released! This trial was called POLARIX and the results demonstrated that polatuzumab vedotin-based treatment…
A recent Phase 3 clinical trial published in Lancet Oncology has identified that camrelizumab used in combination with gemcitabine and cisplatin (chemotherapy) improves progression-free survival (PFS) for those diagnosed with recurrent or…
Findings from a Phase 2 clinical trial for angiosarcoma have just been announced. These findings have demonstrated that the combination of nivolumab and ipilimumab may improve outcomes for patients. This…
Gilles Salles works at the Memorial Sloan Kettering Cancer Center as the Lymphoma Service Chief. She recently sat down to discuss the necessity of new treatments for diffuse large B-Cell…
A recent study investigated the lingering effects of sleep deprivation in healthy adults. This study was conducted by Jeremy Ochab from Poland, alongside his colleagues. Sleep Deprivation Sleep deprivation or…
The FDA has just announced their approval of zanubrutinib (Brukinsa) as a therapeutic option for Waldenström's macroglobulinemia. This approval was given based on the ASPEN Phase 3 clinical trial, and…
A Phase 2 study examining the efficacy of patezolizumab and bevacizumab combined for malignant peritoneal mesothelioma (MPM) patients has just been published in the journal Cancer Discovery. The results demonstrated that…
A recent study conducted by Steven Greenstein evaluated the long-term effects of Epi-off corneal collagen cross-linking (CXL) treatment. This therapy was first FDA approved in 2016. It is a therapy…
New treatments for unresectable hepatocellular carcinoma (HCC) were approved based on a Phase 3 clinical trial. Richard Kim has recently provided an overview of this study. The Study This study ultimately…
Charlene Marshall was diagnosed with idiopathic pulmonary fibrosis (IPF) in 2016. The condition unfortunately has no cure. However, Charlene explains how grateful she is for the scientists that are investigating…
Clinical trial recruitment has always been challenging. It is especially challenging for therapies being tested for rare diseases, an already small population. Clinical trials for rare diseases are small out…
A recent study conducted by researchers at the Broad Institute of MIT and Harvard University uncovered that a small group of cancer cells is responsible for recurrence. Considering that most…
A recent study has uncovered just how many people with pulmonary fibrosis (PF) do not receive an immediate diagnosis. This study conducted in Europe was published in Frontiers of Medicine.…
What is Hyperhidrosis? Hyperhidrosis is the medical term for excessive sweating. Diagnosis is made not necessarily by the amount of sweat, but by the context of when the individual sweats.…
Guadalupe Hayes-Mota has worked for Biogen, Ultragenyx, Amgen, and GSK. He was the prior UCLA Health Director. Additionally, he is a member of the Massachusetts Rare Disease Advisory Council. He…
A recent study investigated the experiences of patients with chronic hypoparathyroidism in pregnancy. The study ultimately found that these patients typically had low risk pregnancies and normal outcomes. However, they…
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