Neuroinflammation is a hallmark of both neuropsychiatric and neurodegenerative conditions and plays a very crucial role in disease progression. In ...

Scientists found a genetic link between autism and DM1, where repeat DNA sequences disrupt brain gene splicing. This sheds light on ASD’s development and opens new paths for targeted treatments.

Researchers suggest discovery of genetic link between ASD and DM1 opens up potential new therapeutic avenues for ASD.

Scientists from The Hospital for Sick Children (SickKids) and the University of Las Vegas, Nevada (UNLV) have uncovered a ...

Health officials in Hood River County have reported an unusual cluster of people coming down with Creutzfeldt-Jakob Disease ... the cases to date. The ‘Molecular Devils’ That Cause the ...

They therefore provide an opportunity to examine pathogenetic mechanisms that underlie the ... diabetes mellitus and accelerated heart disease. If untreated, hormone-excess syndromes can be ...

in the journal Molecular Genetics and Metabolism, highlighting its unique mechanism of action in treating Niemann-Pick disease type C (NPC). The study reveals that arimoclomol enhances the ...

“This elucidation of MIPLYFFA’s ® highly differentiated mechanism of action marks a critical step in understanding its interactions with Niemann-Pick disease type C (NPC) at a cellular level ...

LEXINGTON, Mass. and AMSTERDAM, April 17, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical ...

High dose and low dose AMT-130 demonstrated an 80% and 30% slowing of disease progression vs external control, respectively. The Food and Drug Administration has granted Breakthrough Therapy ...

"Receiving Breakthrough Therapy designation underscores both the urgent need for effective treatments for Huntington's disease and the encouraging interim data demonstrating that AMT-130 has the ...

“Receiving Breakthrough Therapy designation underscores both the urgent need for effective treatments for Huntington’s disease and the encouraging interim data demonstrating that AMT-130 has the ...