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…
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.…
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…
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…
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…
Parent Project Muscular Dystrophy (PPMD) is a nonprofit that focuses on fighting for a cure, better resources, and more awareness for Duchenne muscular dystrophy (DMD). More than a year ago…
Syndax Pharmaceuticals has plans to release data on axatilimab, a treatment for chronic graft-versus-host disease (cGVHD), at the 62nd American Society of Hematology (ASH) Annual Meeting, which will take place…
Edgewise Therapeutics has announced their plan to initiate a Phase 1 trial of EDG-5506, their small molecule therapy for Becker muscular dystrophy. It will take place at Worldwide Clinical Trials…
IntraBio has recently launched an extension study of their Phase II trial of IB1001, a treatment for Niemann-Pick disease Type C. This decision was made shortly after analyzing the positive…
Sierra Oncology has recently released two abstracts that will be presented at the 62nd American Society of Hematology (ASH) Annual Meeting. The meeting is planned for December 5-8. Both abstracts…
Sedor Pharmaceuticals, a partner of Ligand Pharmaceuticals, has recently received FDA approval for SESQUIENT, its status epilepticus treatment. It has been cleared for the treatment of both adult and pediatric…
According to Biospace, Allogene's trial of ALLO-715 for relapsed/refractory multiple myeloma was marred by a patient death. Data from this trial is slated to be presented in December at the…
The entire world has been waiting for a vaccine for COVID-19 since the beginning of the pandemic in March. In response, a number of pharmaceutical companies have entered the race…
The FDA has recently granted the Rare Pediatric Disease (RPD) designation to AMO-02, a treatment for congenital myotonic dystrophy. As there is currently an unmet medical need for this condition,…
BioMarin plans to expand its clinical program for vosoritide, its treatment for achondroplasia. This expansion includes two new phase 2 trials, the first of which will evaluate vosoritide in infants…
Medical professionals are looking towards patient-reported outcomes more and more as a source for research. In fact, a study titled "Patient reported impact of Charcot-Marie-Tooth disease: protocol for a real…
United Therapeutics Corporation has recently presented data from the Phase 3 INCREASE trial, which evaluated Tyvaso as a treatment for pulmonary hypertension associated with interstitial lung disease. Results were presented…
The European Medicines Agency has accepted Biogen's Marketing Authorization Application (MAA) for review. The application is for aducanumab, a treatment for Alzheimer's disease. As this condition faces an unmet medical…
Pancreatic cancer is a rare but severe cancer, and it is often difficult to diagnose until it has advanced. When you combine this with a lack of knowledge and awareness…
Tuberous sclerosis complex (TSC) is a rare disease that, like many other rare diseases, is not yet fully understood by medical professionals. Because of this, it is very difficult to…
The FDA has recently approved of an updated label for Endari, a treatment for sickle cell disease (SCD). This new label will give medical professionals better information, allowing them to…
According to GlobeNewswire, the results from a Phase 1 study of lirentelimab have been released. Allakos developed this medication as a treatment for eosinophilic and mast-cell related diseases. In this…
Researchers from Rensselaer Polytechnic Institute have been conducting research on tau proteins, specifically how it spreads from cell to cell. According to an article in Newswise, this team of researchers…
Currently, there are a number of vaccines for COVID-19 that are being investigated, one of which is AZD1222. The trial was paused back in September when a participant became ill…
Charcot-Marie-Tooth (CMT) disease is a neurological disorder with a number of subtypes. Because of this, no treatment is universally successful within all affected individuals. In order to get patients the…
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