Shionogi launches global Phase 2 trial for late-onset Pompe disease treatment

Esprit is a multicenter, randomized, placebo-controlled, double-blind study assessing the safety, pharmacodynamics, and preliminary efficacy of S-606001 as a substrate reduction therapy

Rhythm Pharmaceuticals’ Phase 3 EMANATE trial falls short of primary goals

The study included four independent genetic substudies targeting obesity caused by heterozygous variants in the POMC/PCSK1, LEPR, SRC1 (NCOA1), and SH2B1 genes

Mankind Pharma acquires Rivotril brand for India

Rivotril is the innovator/ reference brand of clonazepam and is widely prescribed for the management of neurological and psychiatric conditions

IIT Roorkee unveils cutting-edge molecular medicine facility

The facility supports drug discovery, biomolecular research, and the development of next-generation diagnostics

C2N Diagnostics expands Alzheimer’s blood test to South Korea via BeauBrain partnership

Under the agreement, healthcare professionals in the country will gain access to C2N’s PrecivityAD2 blood test

Biogen unveils promising new data for Salanersen in kids with SMA

Salanersen was generally well-tolerated, slowed neurodegeneration, and enabled functional gains

Curatis and Neupharma strike major deal to bring brain tumor therapy to Japan

Neupharma gains exclusive rights to develop and commercialize corticorelin for peritumoral brain edema

Amydis bags $2.5 million NIH grant to develop first eye test for key ALS biomarker

India tackles rare diseases head-on at landmark summit in New Delhi

Industry leaders highlighted the pharmaceutical sector’s role in advancing research, innovation, early screening, and multi-stakeholder collaboration

FDA nod to first treatment for rare genetic brain disorder

The approval comes after a systematic review of published literature