In a recent study entitled “Spinal Muscular Atrophy Phenotype Is Ameliorated Either By SMN Increase Or Modulation Of Secondary Cell Death Events With RNA Therapy (S56.006),” a team of researchers from Milan, Italy, using a stem cell model sought to develop treatment approaches based on RNA for patients with…
A study entitled “Deletion of atrophy enhancing genes fails to ameliorate the phenotype in a mouse model of spinal muscular atrophy,” published in the journal Neuromuscular Disorders, found that removal of genes specifically implicated in muscle atrophy does not improve Spinal Muscular Atrophy phenotype. Spinal muscular atrophy…
Dallas-based biotechnology company AveXis, Inc. announced yesterday that they have successfully dosed the first patient in the intermediate cohort of their Gene Transfer Clinical Trial for chariSM A™ for Spinal Muscular Atrophy Type 1. The news comes just days after the company announced on December 5th that the dosing phase in the low-dose…
Isis Pharmaceuticals announced that it will initiate phase 3 clinical trials to study their investigational treatment for spinal muscular atrophy (SMA), ISIS-SMNRx. The phase 3 study, dubbed CHERISH, will include about 120 non-ambulatory children suffering from the severe and rare genetic neuromuscular disease, and is already the…
Orphan drug development company AveXis Inc. recently announced the completion of the dosing phase in the low-dose cohort of the company’s ongoing clinical trial efforts for its experimental Type 1 spinal muscular atrophy (SMA) therapy. This is the first human gene therapy trial for the disease, which started enrolling participants in late April. SMA is the…
Dallas, Texas-based AveXis, Inc., a biotechnology company working to improve the quality of life of patients suffering from severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA) through the use of gene therapy, is launching a new website specifically dedicated to SMA, called SMAStudy.org. Focused on…
John Carbona, CEO of AveXis, along with Dr. Brian Kaspar have been invited to present the company’s latest developments at the 26th Annual Piper Jaffray Healthcare Conference, which will take place on Tuesday, December 2. AveXis is a biopharmaceutical company currently developing gene therapy-based treatments for severe genetic and orphan diseases such as spinal muscular atrophy (SMA) in order…
Isis Pharmaceuticals, Inc. has announced that it has initiated a Phase 3 clinical trial (CHERISH) that will test the safety and efficacy of their lead drug – ISIS-SMNRx – in non-ambulatory children with spinal muscular atrophy. Spinal Muscular Atrophy (SMA) is a disease caused by mutations in the Smn1 gene that…
Isis Pharmaceuticals, Inc. and AstraZeneca announced a new partnership to develop new, more efficient delivery methods of its antisense oligonucleotides to the desired tissue. Isis is currently utilizing its antisense technology to develop ISIS-SMNRx, an experimental therapy for spinal muscular atrophy being developed in collaboration with Biogen Idec. This new…
PTC Therapeutics, Inc. announced that their collaborative program with Roche and the SMA Foundation (SMAF) for Spinal Muscular Atrophy (SMA) has begun its second phase with adult and children patients, as part of the Phase 1b/2a study. The program is evaluating the safety and tolerability of an under-investigation drug – survival of…
