According to a story from Newswise, a collaborative research team from the University of California San Diego School of Medicine and the Rady Children's Institute for Genomic Medicine will be…
According to a story from PR Newswire, The Autoimmune Registry, Inc. (ARI) has recently released its first ever autoimmune diseases list. This list includes a total of over 150 distinct…
Dilated cardiomyopathy is a heart condition caused by a mutation in the RBM20 gene. This heart condition is unique because it can affect young adults. These individuals are at an…
According to a press release from the biopharmaceutical company Sanofi, the company's investigational treatment avalglucosidase alfa will have its Biologics License Application (BLA) evaluated by the US Food and Drug…
According to an article from Biospace, Takeda Pharmaceuticals has released the results from their Phase 3 HELP trial, an extension study of TAKHZYRO. This hereditary angioedema (HAE) treatment has shown…
PMDD Premenstrual dysphoric disorder (PMDD) is a psychological condition which cyclically occurs with the menstruation cycle. Unfortunately, because the symptoms of this condition can mirror many mental health diagnoses, it…
A recent analysis has shown that eosinophilic gastritis and eosinophilic duodenitis are severely undiagnosed or misdiagnosed as another GI condition. Allakos Inc. has recently reported the results of this study,…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
At the Tufts University School of Medicine, researchers have uncovered a molecular mechanism that eventually leads to a build-up of amyloid beta, which is a major cause of Alzheimer's disease.…
Blavatnik Therapeutics Challenge Awards (BTCA) Harvard Medical School has just announced the winners of their inaugural BTCA. All project winners will receive one million dollars over a period of two…
Lung infections are a common symptom of cystic fibrosis, with Staphylococcus aureus being the most prevalent. While physicians can treat this infection using antibiotics, they do not yet understand its role…
Navigating a Rare Disease in a Pandemic The COVID-19 pandemic has affected everyone, but those already living with a rare disease have unique challenges. With a compromised immune system, they…
Research on myalgic encephalomyelitis, or chronic fatigue syndrome (ME/CFS), has developed in recent years; however, until now there has still been a gap in the literature regarding loneliness. This condition…
Solid Biosciences has just reported that the FDA has finally lifted the hold they had placed on their Phase I/II trial for Duchenne muscular dystrophy (DMD). This trial is called…
Earlier this month, biopharmaceutical company Applied Molecular Transport (AMT) announced the publication of its preclinical data in the Journal of Immunology. You can find the published article here. Within the study,…
Recently, biopharmaceutical company Oncternal Therapeutics ("Oncternal") announced that it has additional clinical data available from its Phase 1 clinical trial. Through the trial, researchers are analyzing an investigational therapy, TK216,…
According to a story from Fierce Pharma, the drug company Alnylam Pharmaceuticals has seen its investigational therapy lumasiran (marketed as Oxlumo) recently gain approval for use in the EU as…
During the Association for the Study of Liver Diseases (AASLD) Liver Meeting, which took place from November 13 to 16, biopharmaceutical company Albireo Pharma ("Albireo) announced data from the Phase…
According to a story from PR Newswire, the pharmaceutical and diagnostics company The Menarini Group recently announced that its drug tagraxofsup (marketed as Elzonris) has a received a positive opinion…
Robert is a 43 year old father of a two year old son named Parker. Robert was diagnosed with inoperable stage 4 bladder cancer about a year and a half…
We often hear people talking about the stress they are feeling during the holidays, while those in the rare disease community silently think, "You have no idea!" During this season,…
According to Charcot-Marie-Tooth News, biotechnology company AcuraStem recently identified 37 potentially therapeutic compounds that could be used to treat patients with Charcot-Marie-Tooth disease type 2A (CMT2A). Currently, CMT2A is the…
The annual meeting of the American College of Rheumatology was recently held, where data was presented on Cimzia and Bimekizumab. Not only were both of these therapies found to benefit…
"Repurposing" drugs means that researchers may discover that certain compounds or therapies for one condition could be translated into the treatment plan for other conditions. Recently, research published in the American…
According to Biospace, Acceleron Pharma has presented preliminary data from their ongoing Phase 2 trial of sotatercept, a treatment for pulmonary arterial hypertension (PAH). The study, titled SPECTRA, is designed…
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