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How the U.S. FDA might respond became a serious question for Wall Street as Sarepta Therapeutics Inc. made known a second death due to acute liver failure with gene therapy Elevidys (delandistrogene ...

The 23andme Holding Co.’s saga came full circle with founder Anne Wojcicki regaining control of the bankrupt genetic testing ...

Calling it “incredible news,” Massachusetts Attorney General Andrea Joy Campbell reported June 16 that U.S. District Judge William Young ordered the Trump administration to restore funding for NIH ...

(NASDAQ:SRPT) plunged to $19.91, down $16.27, or almost 45%, on word of a second death by acute liver failure (ALF) with Elevidys (delandistrogene moxeparvovec), the approved gene therapy for patients ...

Focused on promoting well-being amongst children with neurodivergence, Reset Moves Pty Ltd. created app-based games to help children not only focus with greater clarity, but also to better help them ...

Supernus Pharmaceuticals Inc. is buying Sage Therapeutics Inc. for about $795 million. The deal brings Supernus, already firmly in the CNS market, the only U.S. FDA-approved oral treatment for ...

Nanjing Mingde New Drug Research Co. Ltd. has described potassium voltage-gated channel subfamily KQT member 2/3 (KCNQ2/3) activators reported to be useful for the treatment of epilepsy.

Breakpoint Therapeutics GmbH has divulged Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer.

Myrio Therapeutics Pty Ltd. is set to advance lead product PHOX2B PC-CAR T (PHOX2B peptide-centric chimeric antigen receptor autologous T cells) into clinical trials for relapsed neuroblastoma ...

The U.K. Medicines and Healthcare Products Regulatory Agency is reminding industry that several new regulatory requirements are in effect as of June 16, 2025, such as a postmarket surveillance rule ...

Lung cancer is globally the second most frequent cancer and the cause of approximately 2 of every 10 cancer-related deaths. Immune checkpoint inhibitor therapy can be effective for some patients, but ...