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GSK Duchenne drug gets ‘breakthrough’ status

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The US Food and Drug Administration has granted breakthrough therapy designation to GlaxoSmithKline’s drisapersen for the potential treatment of patients with Duchenne muscular dystrophy. read all at

http://www.pharmatimes.com/Article/13-06-28/GSK_Duchenne_drug_gets_breakthrough_status.aspx

Drisapersen (also known as PRO051 and GSK2402968 ) is an experimental drug under development by Prosensa for the treatment of Duchenne muscular dystrophy.

The compound is in a Phase III trial which is anticipated to complete by the end of 2013.

Drisapersen CAS# 1251830-50-8, has been touted by GlaxoSmithKline and Sarepta as a potential treatment to aid patients with DMD. Results from an early-stage study of drisapersen have been hopeful. Drisapersen is effectual in that it specifically induces exon 51 skipping in the DMD gene. A small, but telling trial of four patients with Duchenne muscular dystrophy offered clinical proof of the effectiveness of drisapersen. In this controlled trial four patients with DMD were administered a single intramuscular 0.8 mg dose of drisapersen. This one dose proved to not only be tolerable and safe, but it also proved to successfully induce exon 51 skipping and dystrophin restoration, up to 35% of normal. The dystrophin restoration was also restored in the majority, up to 94%, of the muscle fibers after the injection. Trials are continuing for the use of drisapersen in treating Duchenne muscular dystrophy in the United States. – See more at: http://www.lgmpharma.com/blog/hope-is-on-the-horizon-for-patients-with-duchenne-muscular-dystrophy/#sthash.oeXyYKc0.dpuf
Drisapersen CAS# 1251830-50-8, has been touted by GlaxoSmithKline and Sarepta as a potential treatment to aid patients with DMD. Results from an early-stage study of drisapersen have been hopeful. Drisapersen is effectual in that it specifically induces exon 51 skipping in the DMD gene. A small, but telling trial of four patients with Duchenne muscular dystrophy offered clinical proof of the effectiveness of drisapersen. In this controlled trial four patients with DMD were administered a single intramuscular 0.8 mg dose of drisapersen. This one dose proved to not only be tolerable and safe, but it also proved to successfully induce exon 51 skipping and dystrophin restoration, up to 35% of normal. The dystrophin restoration was also restored in the majority, up to 94%, of the muscle fibers after the injection. Trials are continuing for the use of drisapersen in treating Duchenne muscular dystrophy in the United States. – See more at: http://www.lgmpharma.com/blog/hope-is-on-the-horizon-for-patients-with-duchenne-muscular-dystrophy/#sthash.oeXyYKc0.dpuf
  1. ^ “PRO051/GSK2402968”. Prosensa. Retrieved 29 October 2012
  2. ^ “A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD114044)”. ClinicalTrials.gov. Retrieved 29 October 2012.

 

Drisapersen CAS# 1251830-50-8, has been touted by GlaxoSmithKline and Sarepta as a potential treatment to aid patients with DMD. Results from an early-stage study of drisapersen have been hopeful. Drisapersen is effectual in that it specifically induces exon 51 skipping in the DMD gene. A small, but telling trial of four patients with Duchenne muscular dystrophy offered clinical proof of the effectiveness of drisapersen. In this controlled trial four patients with DMD were administered a single intramuscular 0.8 mg dose of drisapersen. This one dose proved to not only be tolerable and safe, but it also proved to successfully induce exon 51 skipping and dystrophin restoration, up to 35% of normal. The dystrophin restoration was also restored in the majority, up to 94%, of the muscle fibers after the injection. Trials are continuing for the use of drisapersen in treating Duchenne muscular dystrophy in the United States. – See more at: http://www.lgmpharma.com/blog/hope-is-on-the-horizon-for-patients-with-duchenne-muscular-dystrophy/#sthash.oeXyYKc0.dpuf
Drisapersen CAS# 1251830-50-8, has been touted by GlaxoSmithKline and Sarepta as a potential treatment to aid patients with DMD. Results from an early-stage study of drisapersen have been hopeful. Drisapersen is effectual in that it specifically induces exon 51 skipping in the DMD gene. A small, but telling trial of four patients with Duchenne muscular dystrophy offered clinical proof of the effectiveness of drisapersen. In this controlled trial four patients with DMD were administered a single intramuscular 0.8 mg dose of drisapersen. This one dose proved to not only be tolerable and safe, but it also proved to successfully induce exon 51 skipping and dystrophin restoration, up to 35% of normal. The dystrophin restoration was also restored in the majority, up to 94%, of the muscle fibers after the injection. Trials are continuing for the use of drisapersen in treating Duchenne muscular dystrophy in the United States. – See more at: http://www.lgmpharma.com/blog/hope-is-on-the-horizon-for-patients-with-duchenne-muscular-dystrophy/#sthash.oeXyYKc0.dpuf

1 Comment

  1. medchemnintabelle says:

    Reblogged this on MedCheminAustralia.

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DR ANTHONY CRASTO

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DR ANTHONY MELVIN CRASTO Ph.D

DR ANTHONY MELVIN CRASTO Ph.D

DR ANTHONY MELVIN CRASTO, Born in Mumbai in 1964 and graduated from Mumbai University, Completed his Ph.D from ICT, 1991,Matunga, Mumbai, India, in Organic Chemistry, The thesis topic was Synthesis of Novel Pyrethroid Analogues, Currently he is working with GLENMARK PHARMACEUTICALS LTD, Research Centre as Principal Scientist, Process Research (bulk actives) at Mahape, Navi Mumbai, India. Total Industry exp 30 plus yrs, Prior to joining Glenmark, he has worked with major multinationals like Hoechst Marion Roussel, now Sanofi, Searle India Ltd, now RPG lifesciences, etc. He has worked with notable scientists like Dr K Nagarajan, Dr Ralph Stapel, Prof S Seshadri, Dr T.V. Radhakrishnan and Dr B. K. Kulkarni, etc, He did custom synthesis for major multinationals in his career like BASF, Novartis, Sanofi, etc., He has worked in Discovery, Natural products, Bulk drugs, Generics, Intermediates, Fine chemicals, Neutraceuticals, GMP, Scaleups, etc, he is now helping millions, has 9 million plus hits on Google on all Organic chemistry websites. His friends call him Open superstar worlddrugtracker. His New Drug Approvals, Green Chemistry International, All about drugs, Eurekamoments, Organic spectroscopy international, etc in organic chemistry are some most read blogs He has hands on experience in initiation and developing novel routes for drug molecules and implementation them on commercial scale over a 30 year tenure till date Dec 2017, Around 35 plus products in his career. He has good knowledge of IPM, GMP, Regulatory aspects, he has several International patents published worldwide . He has good proficiency in Technology transfer, Spectroscopy, Stereochemistry, Synthesis, Polymorphism etc., He suffered a paralytic stroke/ Acute Transverse mylitis in Dec 2007 and is 90 %Paralysed, He is bound to a wheelchair, this seems to have injected feul in him to help chemists all around the world, he is more active than before and is pushing boundaries, He has 9 million plus hits on Google, 2.5 lakh plus connections on all networking sites, 50 Lakh plus views on dozen plus blogs, He makes himself available to all, contact him on +91 9323115463, email amcrasto@gmail.com, Twitter, @amcrasto , He lives and will die for his family, 90% paralysis cannot kill his soul., Notably he has 19 lakh plus views on New Drug Approvals Blog in 216 countries......https://newdrugapprovals.wordpress.com/ , He appreciates the help he gets from one and all, Friends, Family, Glenmark, Readers, Wellwishers, Doctors, Drug authorities, His Contacts, Physiotherapist, etc

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