SMA TYPE I , II, III – Clinical Trials with RG7916

Spinraza was the beginning. Roche is in the development of a small molecule named RG7916 a new therapy for SMA. ‘Hope with Roche #SMA #SpinalMuskelAtrofi

Clinical Trials with RG7916 by Roche

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FIREFISH TRIAL FOR SMA TYPE 1
https://www.pioneeringhealthcare.com/sma/clinical-trial-sma-type-1/

A NEW TOOL FOR TRACKING THE PROGRESSION OF SPINAL MUSCULAR ATROPHY (SMA): QUANTITATIVE MAGNETIC RESONANCE IMAGING (QMRI).

https://www.pioneeringhealthcare.com/about/sma/new-tool-tracking-progression-spinal-muscular-atrophy-sma-quantitative-magnetic-resonance-imaging-qmri/

Community Update 08-08-2017
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Community Update 15-08-2017
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Community Update 12-10-2017
Roche released a new community update on the SUNFISH trial for SMA Types 2 + 3
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Community Update 15-3-2018
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SMN protein increased by up to two and a half times. This increase has been sustained throughout the duration of treatment (up to 250 days). To date, RG7916 remains well-tolerated at all doses and no-one has withdrawn from any RG7916 study due to drug related side effects. The information from Part 1 has allowed us to confirm the dose of RG7916 for Part 2 of SUNFISH and the first patient has been enrolled into Part 2.

https://liz-sma-blog.eu/roche-slaepper-stor-uppdatering-pa-de-tva-molekylerna-rg7916-och-olesoxime/

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Firefish Efficacy of RO7034067 in Infants With SMA Type1
Jewelfish A Study of RO7034067
Sunfish A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in SMA Type 2 and 3

First Publication 29. Aug 2016 UPDATED Nils 2018-04-09

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Mia

About Mia

Mamma till tre barn och tvillingarna, en av tvillingarna min äldsta son har SMA Type I-II. Jag arbetar som undersköterska inom akutsjukvården

3 Responses to SMA TYPE I , II, III – Clinical Trials with RG7916

  1. Roches behandlings molekyl RG7916 ökade mängden full längd, funktionellt SMN-protein hos patienter med spinal muskelatrofi

    https://liz-sma-blog.eu/nya-saekerhetsdata-fran-sunfish-del-1-och-molekyl-rg7916-hos-patienter-med-typ-2-eller-3-spinal-muskulatrofi/

    Effects of RG7916 in JEWELFISH, a Study in Patients with Spinal Muscular Atrophy who Previously Participated in a Study with Another SMN2-Splicing Targeting Therapy

    Relationship Between Central and Peripheral SMN Protein Increase Upon Treatment with RO7034067 (RG7916)

  2. Nils says:

    Mechanistic studies of a small-molecule modulator of SMN2 splicing

    Small molecules show great potential for the treat of SMA by rasing SMN protein levels. Researchers have unraveled the cellular mechanisms behind two RG7916-like splice modifiers that increase SMN levels by SMA .

    RG-7916 is a first-in-class drug candidate for the treatment of spinal muscular atrophy (SMA) that functions by modulating pre-mRNA splicing of the SMN2 gene, resulting in a 2.5-fold increase in survival of motor neuron (SMN) protein level, a key protein lacking in SMA patients.

    RG-7916 is currently in three interventional phase 2 clinical trials for various types of SMA. In this report, we show that SMN-C2 and -C3, close analogs of RG-7916, act as selective RNA-binding ligands that modulate pre-mRNA splicing. Chemical proteomic and genomic techniques reveal that SMN-C2 directly binds to the AGGAAG motif on exon 7 of the SMN2 pre-mRNA, and promotes a conformational change in two to three unpaired nucleotides at the junction of intron 6 and exon 7 in both in vitro and in-cell models.