A recent article in BioSpace highlighted presentations by Twist Bioscience, a rapidly growing genomic and synthetic biology company. Twist presented its product line at the virtual 2020 Annual Meeting of…
As reported in Medical Xpress; Fight for Sight, a charity in the UK dedicated to vision, has an exciting announcement for people with macular diseases: they have just granted funding…
There is a certain time of year in which the trees change color, the air becomes crisp and we pour gravy on everything. As I am typing this, I am…
From November 13-16, researchers, doctors, and others participated in the American Association for the Study of Liver Diseases (AASLD)'s Annual Meeting, or The Liver Meeting Digital Experience. During the…
In late November, RNAi therapeutics company Alnylam Pharmaceuticals ("Alnylam") announced that its therapy OXLUMO (Lumasiran) received approval from the European Commission (EC) for both pediatric and adult patients with…
Thanksgiving is a time of gratitude, family and maybe a little gluttony. It was always my favorite time of the year in high school and college. Now, while I still…
When gene therapy company Asklepios BioPharmaceutical, Inc. ("AskBio") and biotechnology company Selecta Biosciences ("Selecta") joined forces to combat genetic disorders, each company brought its best technology and insights. AskBio…
Colette Wheeler is one of four siblings, and although each had only a 50/50 chance of inheriting the genetic mutation that leads to facioscapulohumeral muscular dystrophy (FSHD), all of them…
Takeda Pharmaceuticals has recently awarded XOMA Corporation $2 million for reaching the next milestone in the development of mezagitamab, a treatment for myasthenia gravis (MG). According to Biospace, XOMA has…
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,…
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