Source Cancer Network Several promising drugs for the treatment of lung cancer have been garnering attention over recent years. These new treatments have been highlighted at conferences and are sure to impact the standard of care. Some of these new treatments are detailed below. LOXO-292 Various multikinase inhibitors (MKI) with some activity against RET have
The U.S. Food and Drug Administration (FDA) has granted fast track designation to suvodirsen as a treatment for people with Duchenne muscular dystrophy (DMD). The agency’s decision — which will expedite the review process for suvodirsen — was based on experimental and preclinical data demonstrating the treatment’s potential therapeutic activity. Suvodirsen already had been granted orphan drug designation for the treatment of DMD
Reneo Pharmaceuticals, a clinical stage pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to the company’s lead drug candidate, REN001, for the treatment of fatty acid oxidation disorders (FAOD). “There is a pressing need for effective treatments for patients suffering from
Source The Niche Knoepfler Lab Stem Cell Blog This afternoon Dr. Knoepfler attended and gave public comment at the Medical Board of California stem cell task force meeting on unproven stem cell clinics. There were about 40 people attending. In part this meeting is a consequence of the national governing organization of state medical boards, the Federation of
European Commission, Digital Single Market has published a new Factsheet/Infographic on FET Flagships and Large Research Initiative. These visionary, long-term, large-scale research initiatives address major science and technology challenges in Europe. They involve hundreds of research teams with the aim of boosting the EU’s scientific and industrial landscape.
Source Cell Stem Cell Human pluripotent stem cells (hPSCs) are known to harbor chromosomal aberrations, affecting their tumorigenic potential. We established a strategy to identify cancer-related point mutations in hPSCs, detecting recurrent mutations in over 20 genes, alongside those previously detected in p53. Importantly, naive hPSCs harbor, on average, four times more mutations than their
New interim data from SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age‑appropriate major milestone gain Updated results from global STR1VE study demonstrate that Zolgensma® (onasemnogene abeparvovec-xioi) has significant therapeutic benefit in prolonging event-free survival in SMA Type 1 patients versus natural history Patients in START long-term follow-up study (cohort 2), who
Method is more efficient and economical compared with other procedures A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers. Gene therapy is a clinical technique that introduce genes to treat disease. One approach is to use adeno-associated virus
In new research looking at pivotal trials for new cancer drugs approved by the European Medicines Agency between 2014 and 2016, researchers found only a quarter of trials use overall survival as a primary endpoint. Trials for cancer drugs undertaken in Europe in the mid-2010s were flawed to such a degree as to “raise questions”
In a small safety and feasibility clinical trial, Mayo Clinic researchers have demonstrated for the first time that senescent cells can be removed from the body using drugs termed “senolytics”. The result was verified not only in analysis of blood but also in changes in skin and fat tissue senescent cell abundance. This trial —
