Less than a month after researchers unveiled that a “ticking DNA clock” is behind Huntington’s disease (HD), scientists have ...

Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative ...

Sanofi has entered into a collaboration and license agreement with Alloy Therapeutics to develop an antisense oligonucleotide (ASO) therapy targeting a single, undisclosed central nervous system ...

Cure Rare Disease has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for ...

Spinraza treatment does not lead to significant kidney dysfunction in SMA patients with types 1 and 2, a safety and efficacy ...

Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

Cure Rare Disease launched its SCA3 program in 2021 to develop an antisense oligonucleotide (ASO) therapy, with initial funding support provided by Gregory Klassen, a patient living with SCA3.

to advance the development of an antisense oligonucleotide therapy for spinocerebellar ataxia type 3 (SCA3), a neurodegenerative disorder with no current treatment.