Tags : Duchenne Muscular Dystrophy

Sarepta Reports Two-Year Follow Up Results of SRP-9001 for Duchenne

Shots: Study 101 involves assessing of SRP-9001 (AAVrh74.MHCK7.micro-dystrophin, 2×1014 vg/kg) vs PBO in 4 patients aged 4-7yrs. with DMD The study demonstrated a mean 7.0 point improvement in NSSA score, therapy was well tolerated over 2yrs. time period, @90days. patients had confirmed vector transduction, reduction in CK level SRP-9001 is an investigational gene transfer therapy […]Read More

NS Phrama’s Viltepso (viltolarsen) Receives the US FDA’s Approval for

Shots: The approval is based on P-II two period study; Study 1 includes patients aged 4-10yrs. conducted in North America and Study 2 includes boys aged 5-18yrs. conducted in Japan. The continued approval of VILTEPSO may contingent on confirmation of clinical benefit in P-III confirmatory trial Results: patients receiving the dose of (80 mg/kg/wk), 100% […]Read More

Sarepta Signs an Exclusive Worldwide License with Hansa to Develop

Shots: Hansa to receive $10M up front, up to $397.5M as development, regulatory & commercial milestones. Hansa will book all sales of therapy and will get royalties on gene therapy sales that arises from treating Ab-positive patients enabled through imlifidase pre-treatment Sarepta get an exclusive WW license to develop and promote imlifidase as a pre-treatment […]Read More

PerkinElmer’s Screening Kit Receives the FDA’s Approval as the First

Shots: The US FDA has granted De Novo marketing authorization to PerkinElmer’s GSP Neonatal Creatine Kinase-MM kit based on the clinical study evaluating 3,041 newborns whose dried blood samples showed genetic mutations linked to DMD and the test clinically confirmed 30 samples to have DMD The test act by measuring the level of CK-MM from […]Read More

Sarepta Receives the US FDA’s Approval for VYONDYS 53 (golodirsen)

Sarepta’s VYONDYS 53 is an antisense oligonucleotide derived using phosphorodiamidate morpholino oligomer (PMO) platform targeting DMD patients in patients with a confirmed mutation amenable to exon 53 skipping In Aug 2019, the company received a CRL for VYONDYS 53 from Drug Evaluation 1 following the New Drug Application (NDA) submission to and review by the […]Read More

Santhera Reports Results of Idebenone in SYROS Study for Duchenne

Shots: The SYROS study involves assessing of respiratory function evolution during periods of treatment with idebenone (900 mg/day) (On-Idebenone) compared to period without idebenone treatment (Off-Idebenone) in 18 patients with DMD not using glucocorticoids under Expanded Access Programs following P-III DELOS study, published in Neuromuscular Disorders The SYROS study results demonstrated long-term efficacy in slowing […]Read More

Vertex to Expand its Collaboration with CRISPR Therapeutics to Develop

Shots: Vertex signs a licensing agreement with CRISPR to develop & discover gene editing therapies for DMD and DM1. CRISPR to get $1B, as total deal value including $175M upfront, milestones and royalties on sales. Vertex to get exclusive WW rights for IPR of gene editing therapies including foundational CRISPR/Cas9 technology, novel endonucleases, single and […]Read More

Santhera Reports Results of Raxone (Idebenone) in P-III DELOS trial

Shots: The P-III DELOS trial involves assessing of idebenone (900mg/day) vs PBO in 64 patients with DML for 52 wks. P-III DELOS study results: 50% reduction in annual rates of forced vital capacity; treatment effect remained 6 yrs.; reduction in risk of important patient-relevant outcomes including bronchopulmonary AEs and hospitalizations; reductions in the rate of […]Read More