Maya Doyle is a health social worker specifically focused in pediatrics and the transition from pediatric care to adult care. She has been practicing for twenty years. Now, she works…
The very first National Census of Patients with Rare Diseases in Mexico began October first of 2021 by the General Health Council (CSG). This census will last until January 15th…
Data has recently been announced from a study investigating the efficacy of a new therapy called Alpelisib, for the treatment of PIK3CA-related overgrowth spectrum (PROS). The study was called EPIK-P1.…
Japan's Ministry of Health, Labour, and Welfare has just announced its approval of Alofisel, a stem cell therapy created by Takeda. This approval is specifically for patients who are diagnosed…
CurePSP has just announced their latest Venture Grant recipients. These grants total $320,000 and will all support the research of neurodegenerative disease treatments. CurePSP gives these grants twice every year.…
Emmalyn Reese Hudson was born 11 years ago at 35 weeks gestation weighing in at 5 lbs, 7 ozs. and 18 inches long. Our beautiful baby girl was so tiny…
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…
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