According to a story from Medpage Today, the Society of Hematologic Oncology recently held its virtual meeting on September 9-12. During the event, Dr. Srdan Verstovsek of the MD Anderson…
According to a story from BioSpace, the biopharmaceutical company Bristol Myers Squibb Canada recently announced that its medication fedratinib (marketed as INREBIC®) has been approved by Health Canada as a…
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…
According to a story from PR Newswire, the RNA-targeted gene therapy company Locanabio, Inc. has recently released findings from a preclinical evaluation of an RNA-targeted CRISPR Cas9 (RCas9) system as…
This past week, Cycle Pharma announced FDA approval of a once daily dose of NITYR for patients with hereditary tyrosinemia type 1 (HT-1). According to its prescribing information, NITYR…
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…
Recently, biopharmaceutical company Apellis Pharmaceuticals announced submission of a New Drug Application (NDA) and Marketing Authorization Application (MAA) for pegcetacoplan. If approved, these applications will allow for patients with…
Recently, clinical-stage biopharmaceutical company Aura Biosciences announced a Phase 2 clinical trial to determine the safety, efficacy, and tolerability of AU-011 for patients with choroidal melanoma. Currently, Aura Biosciences…
Several decades ago, gene therapy seemed to be on target to treat a disease by simply replacing a defective gene with a healthy one. A recent article republished in APNews…
Tyrosinemia is a metabolic disorder which prevents the body from processing the amino acid tyrosine. Without treatment, the disorder can be lethal. There are three different types of tyrosinemia with…
According to a recent article in MedicalXpress, in 2004 Professor Sophie Lucas of the Louvain de Duve Institute began studying immunosuppressive cells that are known to block immune responses in…
According to a story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the…
Sarepta Therapeutics has met with the Office of Tissues and Advanced Therapies of the FDA in order to discuss a Type C written response about a clinical trial for SRP-9001,…
Josephine Bila was born with transfusion dependent beta thalassemia, a rare blood disorder. Recently, Josephine uploaded a video to Youtube in which she talks about her life as a patient,…
According to an article from ScienceBlog, a twist on CRISPR gene editing has allowed a team of researchers to successfully treat myotonic dystrophy type 1 in mouse models. By modifying…
Mononine, a treatment for hemophilia B, hit the market nearly three decades ago in 1992. Since then, there have been amazing advancements in the knowledge and treatment for this disease.…
According to a story from BioSpace, the biopharmaceutical company Clene Nanomedicine, Inc., has recently announced that it has completed the process of enrollment patient participants in its upcoming phase 2…
According to a story from BioSpace, the biopharmaceutical company Zogenix, Inc., has recently announced the release of results from a phase 3 clinical trial testing the company's drug fenfluramine (marketed…
According to a story from BioSpace, Genentech (of the Roche Group) has announced its plans to present new data related to its drug satralizumab-mwge (marketed as Enspryng™) at the upcoming…
There is currently an unmet medical need for therapies and research for Parkinson's disease, with the incidence increasing drastically. In fact, the number of cases around the world is expected…
As reported in Science Daily, researchers at the University of Bristol’s MRC Epidemiology Unit working with pharmaceutical companies have developed an approach to reduce the failure rate of developing drugs.…
According to a story from Medscape, thyroid storm is a collection of symptoms that can be described as acute hyperthyroidism (overactivity of the thyroid gland) or thyrotoxicosis, a condition of…
The SLEEP 2020 Annual Meeting will be held virtually due to the COVID-19 pandemic, but that is not stopping Axsome Therapeutics from presenting the results of their Phase 2 CONCERT…
According to a story from BioSpace, the biopharmaceutical company POXEL SA has recently announced that it has commenced with a phase 2 dose ranging clinical trial which will test the…
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