The Royal College of Psychiatrists in the U.K. has published its first-ever guidance to support research into psychedelic drugs as therapies for conditions including treatment-resistant depression, substance abuse disorders and posttraumatic stress disorder, saying that in a fast-moving field there is a risk of jumping ahead of the evidence.
Humanwell Healthcare (Group) Co. Ltd. has divulged P2X purinoceptor 7 (P2RX7; P2X7) antagonists reported to be useful for the treatment of pain, inflammatory, neurological and immunological disorders.
As the first – and so far only – drug to enter clinical testing for the rare neurogenetic disorder Alexander disease, there were some unknowns heading into the readout of the pivotal study testing Ionis Pharmaceuticals Inc.’s zilganersen in children and adults. But the top-line data yielded a clear win for the antisense oligonucleotide candidate, which demonstrated a disease-modifying impact, including statistical significance on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test vs. control at week 61.
Miralogx LLC and Mira Pharmaceuticals Inc. have jointly disclosed new cannabinoid analogues acting as monoamine oxidase-A (MAO-A) and/or MAO-B inhibitors reported to be useful for the treatment of epilepsy, anxiety disorders and pain.
Chinese Academy of Sciences researchers have patented heptanoate compounds reported to be useful for the treatment of amyotrophic lateral sclerosis, Alzheimer’s, Parkinson’s and Huntington’s disease.
Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by the loss of function of the maternally inherited ubiquitin E3 ligase UBE3A gene. AS is characterized by severe symptoms, including lack of speech, epilepsy, developmental and motor skills delays, sleep disturbances and cognitive impairment. Currently, no treatments are available for this disease.
NS Pharma Inc.’s NS-051 (NCNP-04) has been awarded orphan drug designation by the FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping.
Experts agree that the earlier Alzheimer's disease is detected, the sooner action can be taken. And so, the key to preventing deterioration is identifying the most effective early biomarkers, those that can spot the disorder and help halt its progression. Recent advances in the field have pushed a new era of early detection through blood-based biomarkers and personalized medicine strategies based on each patient’s genetic, immunological and clinical profile.
Shanghai Huilun Pharmaceutical Co. Ltd. has synthesized type 2 angiotensin II receptor (AGTR2; AT2) antagonists reported to be useful for the treatment of pain, particularly neuropathic pain.
Vectory Therapeutics BV and Shape Therapeutics Inc. have entered into an option and license agreement granting Vectory an exclusive option to evaluate Shape’s deep brain penetrating AAV capsid, SHP-DB1, for vectorized antibody payloads against three therapeutic targets.
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