According to a recent press release from Dyne Therapeutics, the FDA has placed a clinical hold on their Investigational New Drug (IND) application for DYNE-251. Until this hold is lifted,…
A recent article published in Physician's Weekly highlighted a study that compared four different treatment regimens for lupus nephritis. The options include low-dose cyclophosphamide (LDCyC), high-dose cyclophosphamide (HDCyC), rituximab, and…
Oscar Warrent, a toddler from Norwich, England, has been through a lot in his two years of life. About three months ago, he was diagnosed with rhabdomyosarcoma, a rare form…
We have been living in the midst of a pandemic for years now, and with that has come a number of lasting effects. One of these effects, which has been…
Understanding a rare disease is the first step in treating it. How can you combat the underlying mechanisms of a disorder if you do not know what they are? A…
There are many terms that were once widely used which held offensive origins and meanings towards certain groups. Now, in a more socially conscious society, we are rightfully abandoning these…
Newborn screening (NBS) is a public health service within the United States through which babies are screened for medical conditions that otherwise wouldn't be apparent at birth. Many of the…
Back in December, UCB released the results of its phase 3 trial of rozanolixizumab in adult patients with generalized myasthenia gravis (gMG). Fortunately, these data were positive, pointing to this…
2021 was a hectic, record-breaking, and all around stressful year. Now that it's 2022, I think it's time to start the year with a positive story. According to an article…
Life with a rare disease can be difficult and scary, especially when you can't be sure what the future holds. Jessica Calkins, a 37-year-old mother from Washington, understands this experience…
Most people have heard of Viagra, and they know it as a treatment for erectile dysfunction (ED). Fewer people know it as Revatio, which is indicated for pulmonary arterial hypertension…
Positive results have been announced from a Phase 2 trial of olutasidenib, an investigational selective mIDH1 inhibitor used in combination with azacitidine for the treatment of the mIDH1 form of…
The FDA has recently granted Accelerated Approval to Tarpeyo, also called budesonide, to reduce proteinuria in patients with IgA nephropathy (IgAN). Tarpeyo is the first and currently the only treatment…
The FDA grants the Orphan Drug designation to treatments indicated for rare diseases, which are defined as affecting less than 200,000 people in the U.S. This designation is meant to…
According to a recent press release from uniQure, the first round of observations from a trial investigating AMT-130 for Huntington's disease (HD) have been released. As of now, four patients…
Gareth Crabb is a thoughtful and caring six-year-old boy from Swansea, U.K. He is spending his second Christmas in the hospital this year, as he has anaplastic large cell…
A lack of patient engagement is an issue across many rare diseases, with atypical hemolytic uremic syndrome (aHUS) acting as the perfect example. In fact, aHUS patient organizations are still…
The vaccines for COVID-19 have allowed us to return to normalcy - partially, at least. Now, a UCLA study has discovered that there may be a way to curate a…
According to an article from the National Hemophilia Foundation, CSL Behring and uniQure have recently provided an update on their phase III HOPE-B trial. This study is evaluating etranacogene dezaparvovec,…
Intercept has withdrawn its application for obeticholic acid (OCA) as a treatment for nonalcoholic steatohepatitis (NASH) in Europe. This decision comes after the FDA rejected the company's application in the…
Nick Cannon has announced that he will be taking some time off to be with family and mourn after the death of his son, Zen. The sad news was broken…
Novartis has recently announced the results of their Phase II BYLieve trial, which is investigating Piqray® (alpelisib) as a treatment for hormone-receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-)…
Nobody wants to get sick on vacation; it can ruin a time meant to be fun and relaxing. Unfortunately, Kai McCulloch, a 13-year-old from Scotland, experienced this firsthand. They were…
European patients with recurrent or advanced endometrial carcinoma have a new treatment option, as the European Commission (EC) has approved KEYTRUDA plus LENVIMA. Approval is specifically for adult patients whose…
According to an article posted by Morningstar, IMV Inc. has dosed the first patient in its Phase 1b trial of maveropepimut-S (MVP-S), which will investigate safety in patients with hormone…
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