Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today provided an update on its ongoing open label, multiple dose Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA). To date, all four infants in the 6 mg cohort have been ...
Tags: ISIS, SMA, Infant, respiratory assistance
Plexcera Therapeutics has received approval from Mount Sinai Innovation Partners (MSIP), part of the Icahn School of Medicine, to develop recombinant human acid ceramidase (rhAC) for treatment of diseases caused by genetic or ...
Tags: Plexcera, Cystic Fibrosis
US-based biotechnology firm DART Therapeutics is developing a selective androgen receptor modulator (SARM) drug candidate for Duchenne muscular dystrophy (DMD). DMD is a pediatric rare disease that affects approximately one in 3,600 boys ...
Repligen has announced a new licensing agreement with Pfizer in an effort to advance Repligen's spinal muscular atrophy (SMA) program. The work focuses on RG3039, which is a small molecule drug candidate in clinical development for the ...
Tags: Repligen, Pfizer, spinal muscular atrophy, Medicine
Biogen Idec (BIIB) and Isis Pharmaceuticals have entered into an exclusive, worldwide option and collaboration agreement, pursuant to which the companies will develop and commercialize a novel antisense drug for the treatment of myotonic ...
Tags: Steinert Disease, antisense drug