by Lauren Taylor from In The Cloud Copy Duchenne muscular dystrophy (DMD) is a rare genetic condition that ultimately leads to the wasting away of muscles over time. The condition…
COVID-19 turned our worlds upside down back in March, and it has been the main focus for many medical professionals since. Because it is a novel coronavirus, we did not,…
Editas Medicine has developed a sickle cell disease (SCD) treatment, and it has recently received the Rare Pediatric Disease designation from the FDA. This means that their therapy, EDIT-301, is…
As reported in Crwe World; the biopharmaceutical company Chiasma aims to create oral consumed therapies in order to provide acromegaly patients who have a chronic need for injections with an…
Last week, biopharmaceutical company Deciphera Pharmaceuticals announced results from its Phase 1 clinical trial on QINLOCK (ripretinib). The study, published in the Journal of Clinical Oncology, tested QINLOCK as a potential…
According to an article in the publication Labroots, autism spectrum disorder (ASD) may be the result of interaction between environmental and genetic factors. For instance, researchers point out that ASD…
In our 21st century world, we have become enveloped by technology. In the age of COVID-19, we are even more reliant. In an instant, so many of our every day…
Gary S. Gilkeson Career Development Award (CDA) The CDA is a grant sponsored by the Lupus Foundation of America that was established to help accelerate research for lupus. The 2020…
Living with a rare disease can be difficult. Carlos, who has myositis, knows all about this. His mobility has been declining, and he is now in a wheelchair. His doctors…
In 2012, a new medical technology emerged: CRISPR-Cas9, which offered the opportunity for gene editing. LabBiotech explains that gene editing could benefit patients with genetic disorders by adding, deleting, or…
Myeloproliferative neoplasm (MPN) International Awareness Day is on September 10th. This year, there will be a live streaming event to help bring awareness to this condition, instead of an in-person…
According to MedCity News, a third patient dosed with AT132 during the ASPIRO trial has died. The Phase 1/2 clinical trial tested the efficacy, safety, and tolerability of AT132 for…
Huda Zoghni was born in Lebanon. Huda attended medical school at the American University located in Beirut in 1975. According to an article in Discover, Huda was forced to leave…
BioMarin Pharmaceutical Inc. has recently submitted a new drug application (NDA) for their achondroplasia treatment, vosoritide. This once a day injection is for children who live with the disorder, and…
The World Orphan Drug Conference USA 2020 The Global Orphan Drug Conference and Expo August 24th - August 26th, 2o20 in National Harbor, MD The conference is an event that…
The winning entry for the 2019 Student Voice was published recently in the Orphanet Journal of Rare Diseases. The prize-winning article, a warm and personal account, was written by Annie…
by Caitlin Seida from In The Cloud Copy As if rheumatoid arthritis (RA) weren’t painful enough, those living with the disorder also have to pay extra attention to their hearts…
Kara LeFrance Kara LeFrance is diagnosed with acromegaly, a rare disease, and she's been battling it since she was 20 years old. Unfortunately, it took eight years after her first…
According to Parkinson's News Today, diet is something that should be monitored in Parkinson's disease patients. Many do not eat enough iron, vitamin B1, zinc, or vitamin D, which was…
According to the World Health Organization (WHO), there were 228 million cases of malaria across the globe in 2018. Many of these were in African nations; in fact, the African…
Female mammals have XX chromosomes and males have XY. But scientists have never understood how females decide which of their two X's should be active in each of its cells.…
A study conducted at the University of Texas has discovered that combination therapy may be a viable treatment for cholangiocarcinoma with BRAF mutations. Researchers treated participants with both dabrafenib and…
Dr. Harvey Moldofsky Harvey Moldofsky is a professor emeritus at the University of Toronto. His area of focus is SARS, and since the onset of COVID-19, he has been fully…
Sometimes, researchers find beneficial medications during clinical trials. Other times, the therapy just falls short. According to Myasthenia Gravis News, the latter is true for Firdapse (amifampridine phosphate). The…
by Lauren Taylor from In The Cloud Copy Myasthenia gravis is a neuromuscular disorder characterized by rapid fatigue and weakness of any muscles that are under the voluntary control of…
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