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.

Researchers from Incyte Corp. reported the efficacy of INCB-161734, a KRAS G12D inhibitor, in combination with various strategies in models of pancreatic ductal adenocarcinomas (PDACs).

At the ESMO Targeted Anticancer Therapies Congress held this week in Paris, researchers at Mabqi SAS presented the preclinical characterization of MQI-201, an anti-TRPV6 therapeutic antibody in models of metastatic prostate cancer.

A new way of understanding Alzheimer’s disease, based on biological inflection points that mark decisive moments in the progression of the disorder, could change how new drugs are developed to achieve more effective therapies. This new perspective could rethink strategies that depend not so much on the target itself, but on the precise moment at which it is addressed.

Exon skipping therapies based on antisense phosphorodiamidate morpholino oligomer (PMO) have great potential to restore dystrophin in the skeletal muscle and treat Duchenne muscular dystrophy (DMD). Entrada Therapeutics Inc. has developed an endosomal escape vehicle conjugated to DMD exon skipping PMOs (exon 51 skipping), ENTR-601-51, for the potential treatment of DMD.

Neurodegenerative disease and cognitive decline cannot be explained by a single process. Beta-amyloid plaques, hyperphosphorylated tau, alpha-synuclein, activated microglia and astrocytes, altered receptors such as TREM2, mitochondrial dysfunction, epigenetic changes and cerebrovascular alterations all seem to contribute to the development of dementia in Alzheimer’s disease (AD). While scientists attempt to address each of these elements, prevention is growing as a primary goal.

Facioscapulohumeral muscular dystrophy (FSHD) is a muscle wasting disease caused by aberrant expression of double homeobox protein 4 (DUX4). When DUX4 is activated in skeletal muscle, it triggers myocyte cell death after several transcriptional changes, thus genetic DUX4 silencing arises as a promising approach for treating FHSD.

Excellergy Inc. has presented data for Exl-111, an investigational allergic effector cell response inhibitor (ECRI) targeting the IgE axis that is designed to disarm allergic effector cells at the source of activation.