According to a story from the AKU Society, the European Medicines Agency (EMA) has recently announced a positive opinion on extending the label indication for nitisinone (marketed as Orfadin®). The…
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,…
Recently, Blueprint Medicines Corporation ("Blueprint") shared that the European Commission (EC) approved marketing authorization of AYVAKYT (avapritinib). The therapy is designed to treat adult patients with PDGFRA D842V mutated gastrointestinal stromal…
Early last week, biopharmaceutical company MediciNova Inc. ("MediciNova") announced promising results for their therapeutic candidate MN-166 (ibudilast). The company, which published in the findings in Cancer Chemotherapy and Pharmacology, explored…
Last week, biotechnology company Anokion SA ("Anokion") announced the FDA's acceptance of an Investigational New Drug (IND) application. Anokion submitted the IND for ANK-700, a therapy for patients with…
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…
Arrowhead Pharmaceuticals recently announced positive twenty-four-week biopsy results from four patients who participated in the first cohort of the Phase II clinical trial of ARO-AAT. ARO-AAT is Arrowhead’s investigational…
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…
On September 25, biotechnology company Vertex Pharmaceuticals Incorporated ("Vertex") announced FDA-approval of KALYDECO (ivacaftor) for pediatric patients with cystic fibrosis (CF). Specifically, KALYDECO can treat infants between 4-6 months…
Recently, research and development (R&D) company Neurophth Therapeutics announced that the FDA granted Orphan Drug designation to its therapy NR082. Also known as rAAV2-ND4 or NFS-01 project, NR082 is…
According to a recent press release, Japan just approved ULTOMIRIS (ravulizumab) for patients with atypical hemolytic uremic syndrome (aHUS). Developed by biopharmaceutical company Alexion Pharmaceuticals ("Alexion"), ULTOMIRIS represents a…
In a recent press release, precision therapy company Blueprint Medicines Corporation announced positive results from two clinical trials: EXPLORER and PATHFINDER. The trials examined the safety, efficacy, and tolerability…
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,…
According to a recent article in Business Wire, it is estimated that five million people in the United States over the age of sixty-five are coping with Alzheimer’s at…
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…
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