Mutations in the NF1 gene lead to neurofibromatosis type 1, which often leads to bone abnormalities and spine deformity. Efforts to treat or even prevent the disease have been stymied by lack of understanding about how the disease occurs and progresses.
In October, the Nobel Committee awarded the 2025 Nobel Prize in Physiology or Medicine to Shimon Sakaguchi, Mary Brunkow and Fred Ramsdell for their discoveries in the field of autoimmunity. As has become typical for the scientific Nobel Prizes, the award-winning research is by now several decades old. But the discoveries were the basis for ongoing research into how to prevent autoimmunity that notched significant wins in 2025, in both basic research and in the clinic.
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Laekna Pharmaceutical Ningbo Co. Ltd. has identified Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer.
Shanghai Xianxiang Medical Technology Co. Ltd. has synthesized proteolysis targeting chimera (PROTAC) compounds comprising a cereblon (CRBN)-binding moiety coupled to an EGFR (HER1; erbB1) mutant-targeting moiety through a linker reported to be useful for the treatment of cancer.
Eilean Therapeutics LLC has presented data for ZE74-0282, a novel small molecule that targets the JAK2 JH2 domain harboring the V617F mutation with no impact on wild-type JAK2.
Bronchopulmonary dysplasia (BPD) remains a significant challenge in neonatal care, particularly affecting preterm infants with low birth weight who often require oxygen therapy or mechanical ventilation to survive.
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