Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

GSK’s antisense-based drug for chronic hepatitis B (CHB), bepirovirsen, will get a rapid six-month review from the FDA when it is filed for approval, said the drugmaker this morning. The fast ...

The Centers for Medicare and Medicaid Services is directing all Medicare Advantage plans to cover Qalsody for treating ...

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MDA announces over $5 million in grants to advance neuromuscular disease research, including 14 research grants and 7 ...

Dec. 6, 2024 — Researchers have unraveled how immune cells called microglia can transform and drive harmful processes like neuroinflammation in Alzheimer's disease. ...

INOVIQ (ASX:IIQ) has completed a proof-of-concept (POC) study showing that exosomes—tiny vesicles released from cells—can be ...

San Diego Symphony’s “Noel, Noel,” Dea Huston’s “Black Family Christmas,” the Santacon festival, Eve Selis returns and more ...

Metabolic dysfunction-associated steatotic liver disease (MASLD) affects around 30% of the global population. Studies suggest ...

When Anna moved in, her apartment had no oven, fridge, or washing machine; the sink wasn’t usable; and the kitchen cabinets were totally broken. “But the ceilings, French doors, and floors were so ...

SRP-5051 is an antisense oligonucleotide commercialized by Sarepta Therapeutics, with a leading Phase II program in Duchenne Muscular Dystrophy.