Ractigen Therapeutics Co. Ltd. has entered into a formal collaboration with University Medical Center Utrecht, affiliated with Utrecht University, to advance the development of small activating RNA (saRNA)-based treatments for a spectrum of intractable neurodevelopmental disorders.
Investigators have identified five cases of so-called iatrogenic Alzheimer’s disease (AD), that is, AD that was acquired as a result of undergoing medical procedures. A team led by University College London scientists reported their findings online in Nature Medicine on Jan. 29, 2024.
Sarepta Therapeutics Inc.’s next-generation peptide-conjugated PMO therapy, SRP-5051 (vesleteplirsen), looks set to stake its claim in the Duchenne muscular dystrophy (DMD) space, as phase II data unveiled dystrophin expression that outperforms first-generation exon-skipping drug Exondys 51 (eteplirsen). The question is what that space might look like in the wake of a U.S. FDA decision whether to expand labeling and convert to full approval Sarepta’s DMD gene therapy, Elevidys (delandistrogene moxeparvovec).
In its first patenting, Cambridge, U.K.-based Opto Biosystems Ltd. is seeking protection for implantable sensors that may be used in systems to measure chemical, biological, or electrical signals in the central and/or peripheral nervous systems.
After almost 10 years on the market, the EMA is to withdraw its conditional approval of the Duchenne muscular dystrophy treatment Translarna (ataluren), after concluding there is still not enough evidence of its effectiveness.
Researchers from the University of Maryland Baltimore and University of Maryland Baltimore County are seeking patent protection for an angle-tuned (AT) ring coil for improving the depth-spread performance of transcranial magnetic stimulation (TMS) coils as well as high-performance composite coils and multisite TMS systems.
An international working group has proposed a new research framework that stages Parkinson's disease (PD) and defines it based on the disease's underlying biology, rather than on clinical symptoms.
The discovery of how proteins fold and what happens if they don’t, or do it wrong, has impacted medicine. Protein function and several pathologies depend on folding. Four scientists, Franz-Ulrich Hartl, Arthur Horwich, Kazutoshi Mori and Peter Walter, revealed the role of chaperones in assisting proteins to acquire their correct structure and described a failure warning system, the unfolded protein response (UPR). For their studies, the researchers will receive the 2024 Frontiers of Knowledge Award in Biology and Medicine from the BBVA Foundation.
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