Sound Pharmaceuticals announced the first-ever Phase III clinical trial protocol for Meniere’s disease to be approved by the FDA. Meniere’s disease (MD) is a neurotologic disorder that involves tinnitus (ringing…
A study in the Journal of Movement Disorders on patients with Parkinson's disease has found a relationship between sedentary living and attention difficulty. Exercise is the age old medicine, with…
A recent study has demonstrated that through standardizing the whole genome sequencing process and making it routine for patients, we can improve rare disease diagnosis across the board. Earlier diagnosis…
Emily Ventura was already well aware of the risk of germs before coronavirus. She works as a nurse in the ICU and takes care of her 8-year-old, who is immunosuppressed.…
According to a recent article in The Guardian, funding received from the NIHR and Medical Research Council will facilitate the launch of the largest genetic study of chronic fatigue syndrome…
uniQure has recently began their Phase I/II clinical trial of AMT-130, a gene therapy for the treatment of Huntington's disease. Two patients are included so far, with one being given…
Researchers believe that they may have found the cause of myasthenia gravis (MG), according to an article in Myasthenia Gravis News. A study found that issues with mitophagy in immune…
Global Blood Therapeutics (GBT) will submit their Marketing Authorization Application (MAA) for Oxbryta to the EMA by 2021, says a recent BioSpace press release. Ultimately, the MAA will seek regulatory…
Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced that the United States Food and Drug Administration (FDA) has granted…
Why are most people able to tolerate cat’s hair while some people have severe reactions to the ubiquitous and practically invisible dust mites? A recent SciTech Daily article followed La…
Ultragenyx Pharmaceutical's therapy, CRYSVITA, has recently been approved by the FDA for the treatment of FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors. This approval comes after…
According to a story from Cystic Fibrosis News Today, Eloxx Pharmaceuticals had just announced recently that it is officially re-opening enrollment for its phase 2 clinical study. This trial will…
According to LabioTech, a psoriasis drug developed by biotechnology company Affibody showed promising results in a Phase II clinical trial (AFFIRM-35). This trial, which consisted of 108 participants with…
According to a story from ANCA Vasculitis News, a recent review study has determined that patients living with ANCA vasculitis and other related diseases are at a higher than average…
For most drug therapies, researchers test on animal models of a disease before testing on humans. Recently, using microRNA-483, a small RNA molecule, on rat models of PAH reduced…
Happy Friday! The world could always use more good news, and this week we've got some new discoveries to discuss. Up first, details on a trial that may have finally…
As you know, COVID-19 has fundamentally changed our daily life. What began as a novel coronavirus in Wuhan, China has developed into a global pandemic: 9 million diagnosed cases…
In a Business Wire press release, the Canadian NASH Network (CanNASH) shared the results of a recent study predicting a severe increase in cases of nonalcoholic fatty liver disease…
COVID-19 has now taken hold in over one hundred eighty countries. According to a recent article in The Lancet, in May of 2020, the United States reported the highest…
As reported in Business Fortnight, the top level gene therapy company AVROBIO has announced successful clinical trials for investigational drugs for Fabry disease and cystinosis. The CEO of AVROBIO, Geoff…
The Rare Diseases Clinical Research Network (RDCRN) has recently launched a survey aiming to assess the impact of COVID-19 on the rare disease patient community. This initiative is important as…
Cures Within Reach is an organization that aims to help patients by repurposing research. They work towards getting treatments quickly and safely in an effort to aid those who need…
According to a story from BioSpace, the biopharmaceutical company Dicerna Therapeutics, Inc. has announced recently that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to…
Earlier this year, The Leukemia & Lymphoma Society hosted a presentation by Dr. Eunice Wang, Clinical Leukemia Service, Professor of Oncology, Roswell Park Comprehensive Cancer Center. Dr. Wang’s presentation…
If you're like me, your life has changed immensely over the past few months. COVID-19, a novel coronavirus with 8.39 million diagnoses worldwide, has kept us inside and changed…
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