Researchers from Arthex Biotech SL, the University of Valencia (Spain) and collaborators sought to address the lack of optimized microRNA (miRNA)-targeting antisense oligonucleotides (anti-miRs) for upregulating MBNL1 in myotonic dystrophy.
A new Danish research project focused on Parkinson’s disease has received funding from Innovation Fund Denmark. The DESYNA (Degradation of Extracellular α-SYNuclein Aggregates) project aims to develop a new therapy targeting α-synuclein, the accumulation of which is a key driver of Parkinson’s disease.
The aberrant activation of NACHT, LRR and PYD domains-containing protein 3 (NLRP3) inflammasome is a known crucial driver of neuroinflammation in Alzheimer’s disease (AD) and other related dementias. The inhibition of NLRP3 has shown benefits in preclinical models. Researchers from the University of Texas recently presented data regarding an NLRP3 inhibitor, AMS-17, for the treatment of AD and related dementias.
Parkinson’s disease (PD) involves the progressive loss of dopaminergic neurons, particularly in the substantia nigra. This neurodegeneration is linked to the abnormal accumulation of α-synuclein, a protein that forms toxic aggregates and spreads between cells, damaging them. At the 20th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD), held from March 17 to 21, 2026, in Copenhagen, several strategies were presented that aim to modify the course of the disease and offer real alternatives to purely symptomatic treatments.
Suven Life Sciences Ltd. has disclosed new 2-amino-pyrimidine derivatives acting as muscarinic M4 receptor positive allosteric modulators (PAMs) reported to be useful for the treatment of pain, sleep disorders, depression, schizophrenia, Alzheimer’s, Parkinson’s and Huntington’s disease.
Congruence Therapeutics Inc. has closed a $39.5 million financing to advance into its portfolio of small-molecule correctors for diseases of protein misfolding into the clinic.
At the ongoing International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD 2026) in Copenhagen, researchers presented details on a new SERCA inhibitor from Synuca Therapeutics ApS – SYN-4569 – as a potential approach to treat multiple system atrophy-cerebellar type (MSA-C).
Shanghai Huilun Pharmaceutical Co. Ltd. has synthesized potassium voltage-gated channel subfamily KQT member 2/3 (KCNQ2/3) activators. They are reported to be useful for the treatment of epilepsy, neuropathic pain, ischemic stroke and neurodegeneration.
Biohaven Therapeutics Ltd. has presented prodrugs of betahistine reported to be useful for the treatment of obsessive-compulsive disorder, trichotillomania, Tourette syndrome, amyotrophic lateral sclerosis, multiple sclerosis and more.
Researchers from the China Pharmaceutical University and Guangdong Pharmaceutical University (China) have unveiled the crucial role of the alternative splicing of E2A in myogenic progression and demonstrated that PTBP1, by controlling E2A alternative splicing, is a critical regulator of myogenesis.
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