Fulcrum Therapeutics is beginning a Phase 1 trial of FTX-6058, a treatment for sickle cell disease (SCD). Researchers will focus on the tolerability, safety, and pharmacokinetics of the medication. The…
As written in BioSpace, Genetech has recently presented new two-year data on their spinal muscular atrophy (SMA) treatment, evrysdi. The information was released at the 25th International Annual Congress of…
The Annals of the American Thoracic Society have recently released an article that evaluates how the COVID-19 pandemic disproportionately affects those who have already dealt with health inequities throughout history,…
The FDA has recently granted two designations, the rare pediatric disease and orphan drug designations, to Spirovant Sciences' gene therapy for cystic fibrosis (CF). SPIRO-2101 will now be able to…
Nate Peters is an air force veteran, and he also lives with ankylosing spondylitis. This means that he knows what it's like to spend a lot of time in the…
Eisai has announced that they will begin a Phase 3 trial, titled MOMENTUM, to evaluate lorcaserin as a treatment for Dravet syndrome. This decision comes after a consultation with the…
Seelos Therapeutics and Duke University have both signed a Sponsored Research Agreement, meaning they will work in tandem to evaluate and test SLS-004, a Parkinson's disease treatment. Their research aims…
October is Rett Syndrome Awareness Month, and even though the pandemic has changed the way that we celebrate it, there are plenty of ways to get involved! Rettsyndrome.org has organized…
A study reported in NewsWise evaluates retinoid-x receptor (RXR) agonists as a treatment for recurring and refractory multiple sclerosis (MS). This specific RXR agonist, bexarotene, was found to help remyelination,…
According to an article from BioSpace, the TargetCancer Foundation has began enrolling patients in their TRACK study. This study is intended to provide rare cancer patients with individualized treatment options…
According to an article from BioSpace, Vivet Therapeutics and Pfizer have entered into a manufacturing agreement to develop Vivet's gene therapy, which treats Wilson disease. The terms of the agreement…
Vertex Pharmaceuticals will present data on the long-term use of their cystic fibrosis (CF) treatments at the 43rd European Cystic Fibrosis Digital Conference, and then again at the 2020 North…
APT-1011 is a treatment for eosinophilic esophagitis, a condition that currently faces an unmet medical need. The medication was originally developed by TPG Capital, but they created a new company,…
Medical professionals have already discovered a number of genes that play a role in pulmonary arterial hypertension (PAH), but according to LabRoots, they have found a new one. The ABCC8…
According to an article from Charcot-Marie-Tooth News, a study was conducted that discovered three new mutations on the GJB1 gene that result in X-linked Charcot-Marie-Tooth disease (CMTX1). The study, which…
BioMarin has been working to develop treatments for phenylketonuria for 15 years, and it is a cause they are very committed to. That is why they are so excited to…
Lupus Canada and the Lupus Foundation of America have joined together to award the 2020 Lupus Canada Catalyst Award. This grant is intended to fund one year of research that…
When children have problems at school, it is often linked to something bigger going on in another aspect of their life, whether that is at home or with friends. A…
CRISPR Therapeutics, in combination with Vertex Pharmaceuticals, has recently announced that the European Medicines Agency (EMA) has granted the Priority Medicines (PRIME) designation for their sickle cell disease (SCD) treatment,…
Chronic pain is a common problem in the United States; it is estimated that 25.3 million people in the US experience chronic, severe pain every day. Within these millions of…
A new treatment for acromegaly has recently become available for patients. Called MYCAPSSA, this delayed-release capsule is intended for long term maintenance of acromegaly. Considering that injections are the typical…
According to an article from BioTech 365, data from the Phase 3 INVICTUS trial and Phase 1 dose-escalation study, which evaluated QINLOCK as a treatment for gastrointestinal stromal tumors, was…
Cynata Therapeutics Limited has recently announced the results from their preclinical trial of induced pluripotent stem cell (iPSC)-derived Cymerus™ mesenchymal stem cells (MSCs), according to BioSpace. They are testing these…
Jazz Pharmaceuticals has launched an educational program called Find the Right Fit, which aims to spread awareness and help patients with myelodysplastic syndromes (MDS) and secondary acute myeloid leukemia (sAML).…
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Dr. Joan T. Merrill is a professor of medicine at the University of Oklahoma who presented on lupus at the 2020 Congress of Clinical Rheumatology - East. She spoke about…
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