When the drug fomivirsen was approved by the FDA in 1998 for the treatment of cytomegalovirus retinitis in patients with HIV/AIDs, it was hailed as a milestone in drug discovery because it was the ...

Patients treated with high-dose BIIB080 saw positive trends on the global Clinical Dementia Rating Sum of Boxes, Mini-Mental State Exam cognitive scales and Functional Activities Questionnaire at week ...

Researchers have found that a promising stroke therapy, known as antisense oligonucleotides, is preferentially taken up from the blood into areas of stroke damage in the brain when the molecules are ...

HRN001 is a potent and specific antisense oligonucleotide designed to drive controlled upregulation of FAN1, a genetically validated ...

CAMBRIDGE, Mass., April 02, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BIIB080, an ...

SEATTLE, May 16, 2023 (GLOBE NEWSWIRE) -- Chinook Therapeutics, Inc. (KDNY) today announced it has entered a collaboration agreement with Ionis Pharmaceuticals, Inc. for the discovery, development and ...

Diffuse intrinsic pontine glioma (DIPG) is a lethal pediatric brain cancer that often kills within a year of diagnosis. DIPG occurs in an area of the brainstem called the pons, which controls many of ...

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the ...

Please provide your email address to receive an email when new articles are posted on . Phase 1b trial of BII080 demonstrated dose-dependent and sustained reduction of tau protein in CSF, as well as ...

An experimental GSK drug in development for treating chronic hepatitis B now has results from two pivotal studies showing the therapy brought levels of the virus low enough to allow the immune system ...

Dravet Syndrome is a rare, catastrophic, lifelong form of epilepsy that begins in a baby’s first year of life with frequent and/or prolonged seizures. About 1 in 16,000 people are affected by Dravet ...

Having a rare genetic disease is actually pretty common. Rare diseases affect approximately 1 in 10 individuals, and more than 30 million people in the U.S. have a rare disease diagnosis. What makes ...