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 [email protected], 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. [18][19] Subsequently, nusinersen was approved to treat SMA in Canada (July 2017),[20] Japan (July 2017),[21] Brasil (August 2017),[22] and Switzerland (September 2017). ヌシネルセンナトリウム The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. Each 1 mL solution contains 2.4 mg of nusinersen (equivalent to 2.53 mg of nusinersen sodium salt). Toxicity in the nervous system (neurotoxicity) was observed in animal studies.

Spinraza is an injection administered into the fluid surrounding the spinal cord. [17], In October 2017, the authorities in Denmark recommended nusinersen for use only in a small subset of people with SMA type 1 (young babies) and refused to offer it as a standard treatment for all other people with SMA quoting an "unreasonably high price" compared to the benefit. The FDA granted this application fast track designation and priority review. C234H323N61Na17O128P17S17 : 7500.89 [2] The primary route of elimination is likely by urinary excretion for nusinersen and its metabolites. [16][17] It was approved by the FDA in December 2016 and by EMA in May 2017 as the first drug to treat SMA. The drug is metabolized via exonuclease (3’- and 5’)-mediated hydrolysis and does not interact with CYP450 enzymes. It is administered directly to the central nervous system (CNS) using intrathecal injection. [5], The drug is used to treat spinal muscular atrophy associated with a mutation in the SMN1 gene. Patients were randomized to receive an injection of Spinraza, into the fluid surrounding the spinal cord, or undergo a mock procedure without drug injection (a skin prick). [7], The drug distributes to CNS and peripheral tissues. [25], In August 2018, the National Institute for Health and Care Excellence (NICE), which weighs the cost-effectiveness of therapies for the NHS in England and Wales, recommended against offering nusinersen to people with SMA. The drug is administered directly to the central nervous system using intrathecal injection once every 3–4 months. Sorry, your blog cannot share posts by email. Abametapir アバメタピル , абаметапир , أباميتابير , 阿巴甲吡 , Drug Scaleup and Manufacturing International, rare pediatric disease priority review voucher, adverse effects from the spinal injection, University of Massachusetts Medical School, “International Nonproprietary Names for Pharmaceutical Substances (INN). Patients survive owing to low amounts of the SMN protein produced from the SMN2 gene. Spinraza is an injection administered into the fluid surrounding the spinal cord. Nusinersen modulates alternate splicing of the SMN2 gene, functionally converting it into SMN1 gene, thus increasing the level of SMN protein in the CNS. [2], In clinical trials, the drug halted the disease progression. Oat Straw (Avena sativa) helpful in calming the nerves of those who are detoxing from drug or alcohol addiction, and can even help curb nicotine cravings. In around 60% of infants affected by type 1 spinal muscular atrophy, the drug also significantly improved motor function. 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. etc in organic chemistry are some most read blogs He has hands on experience in initiation and developing novel routes for drug molecules [11], Starting in 2012, Ionis partnered with Biogen on development and in 2015 Biogen acquired an exclusive license to the drug for a US$75 million license fee, milestone payments up to US$150 million, and tiered royalties thereafter; Biogen also paid the costs of development subsequent to taking the license. Sorry, your blog cannot share posts by email. Additional open-label uncontrolled clinical studies were conducted in symptomatic patients who ranged in age from 30 days to 15 years at the time of the first dose, and in presymptomatic patients who ranged in age from 8 days to 42 days at the time of first dose. [26] Children with SMA type 1 were treated in the UK under a Biogen-funded expanded access programme; after enrolling 80 children, the scheme closed to new people in November 2018. [24], Norwegian authorities rejected the funding in October 2017 because the price of the medicine was "unethically high". 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. [2], Spinal muscular atrophy is caused by loss-of-function mutations in the SMN1 gene which codes for survival motor neuron (SMN) protein. His friends call him Open superstar worlddrugtracker. [2], The half-life is estimated to be 135 to 177 days in CSF and 63 to 87 days in blood plasma. Fill in your details below or click an icon to log in: You are commenting using your WordPress.com account. In December 2016, it became the first approved drug used in treating this disorder. [22] Norwegian authorities rejected the funding in October 2017 because the price of the medicine was “unethically high”. Nusinersen,[1] marketed as Spinraza,[3] is a medication used in treating spinal muscular atrophy (SMA),[4] a rare neuromuscular disorder. [27] In May 2019, however, NICE reversed its stance and announced its decision to recommend nusinersen for use across a wide spectrum of SMA for a 5 year period. [2], Nusinersen is an antisense oligonucleotide in which the 2’-hydroxy groups of the ribofuranosyl rings are replaced with 2’-O-2-methoxyethyl groups and the phosphate linkages are replaced with phosphorothioate linkages. In January 2018 public funding of Spinraza was approved in Israel.

[1], Although not observed in the trial patients, a reduction in platelets as well as a risk of kidney damage are theoretical risks for antisense drugs and therefore platelets and kidney function should be monitored during treatment.

Spinraza is approved for use across the range of spinal muscular atrophy patients.

ISIS-SMNRx is a drug that is designed to modulate the splicing of the SMN2 gene to significantly increase the production of functional SMN protein.