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Mavoglurant (AFQ-056) is an experimental drug candidate for the treatment of fragile X syndrome

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マボグルラントFile:Mavoglurant.svg

ماووگلوران

Mavoglurant (AFQ-056) is an experimental drug candidate for the treatment of fragile X syndrome.[1] It exerts its effect as an antagonist of the metabotropic glutamate receptor 5 (mGLU5).[2]

Mavoglurant is under development by Novartis and is currently in Phase II and Phase III clinical trials.[1][3] If successful, it would be the first drug to treat the underlying disorder instead of the symptoms of fragile X syndrome.[4]

 

  1. P. Cole (2012). “Mavoglurant”. Drugs of the Future 37 (1): 7–12. doi:10.1358/dof.2012.37.1.1772147. 
  2. Levenga, J; Hayashi, S; De Vrij, FM; Koekkoek, SK; Van Der Linde, HC; Nieuwenhuizen, I; Song, C; Buijsen, RA et al. (2011). “AFQ056, a new mGluR5 antagonist for treatment of fragile X syndrome”. Neurobiology of disease 42 (3): 311–7. doi:10.1016/j.nbd.2011.01.022. PMID 21316452. 
  3.  Jacquemont, S.; Curie, A.; Des Portes, V.; Torrioli, M. G.; Berry-Kravis, E.; Hagerman, R. J.; Ramos, F. J.; Cornish, K. et al. (2011). “Epigenetic Modification of the FMR1 Gene in Fragile X Syndrome is Associated with Differential Response to the mGluR5 Antagonist AFQ056”. Science Translational Medicine 3 (64): 64ra1. doi:10.1126/scitranslmed.3001708. PMID 21209411. 
  4.  “AFQ056 drug improves symptoms in Fragile X patients: Study”. news-medical.net. January 9, 2011.

Fragile X syndrome (FXS), Martin–Bell syndrome, or Escalante’s syndrome (more commonly used in South American countries), is a genetic syndrome that is the most widespread single-gene cause of autism and inherited cause of mental retardation among boys. It results in a spectrum of intellectual disabilities ranging from mild to severe as well as physical characteristics such as an elongated face, large or protruding ears, and large testes (macroorchidism), and behavioral characteristics such as stereotypic movements (e.g. hand-flapping), and social anxiety.

Fragile X syndrome is associated with the expansion of the CGG trinucleotide repeat affecting the Fragile X mental retardation 1 (FMR1) gene on the X chromosome, resulting in a failure to express the fragile X mental retardation protein (FMRP), which is required for normal neural development. Depending on the length of the CGG repeat, an allele may be classified as normal (unaffected by the syndrome), a premutation (at risk of fragile X associated disorders), or full mutation (usually affected by the syndrome).[1] A definitive diagnosis of fragile X syndrome is made through genetic testing to determine the number of CGG repeats. Testing for premutation carriers can also be carried out to allow for genetic counseling. The first complete DNA sequence of the repeat expansion in someone with the full mutation was generated by scientists in 2012 using SMRT sequencing.

There is currently no drug treatment that has shown benefit specifically for fragile X syndrome. However, medications are commonly used to treat symptoms of attention deficit and hyperactivity, anxiety, and aggression. Supportive management is important in optimizing functioning in individuals with fragile X syndrome, and may involve speech therapy, occupational therapy, and individualized educational and behavioral programs.

orphan drug designation EMA

http://www.google.co.in/url?sa=t&rct=j&q=&esrc=s&frm=1&source=web&cd=3&cad=rja&ved=0CDwQFjAC&url=http%3A%2F%2Fwww.ema.europa.eu%2Fema%2Findex.jsp%3Fcurl%3Dpages%2Fmedicines%2Fhuman%2Forphans%2F2012%2F11%2Fhuman_orphan_001121.jsp%26mid%3DWC0b01ac058001d12b&ei=OyzzUZvtN8izrgf-4YCwDA&usg=AFQjCNHkQH6bIGW8MtvFbViHQ5mLeLRmFQ&sig2=BqsRq2kZF3lgbKix5Qji5g&bvm=bv.49784469,d.bmk

EU/3/12/1046: Public summary of opinion on orphan designation: Mavoglurant for treatment of fragile-X syndrome

http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2012/11/human_orphan_001121.jsp&mid=WC0b01ac058001d12b

http://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=317238

Week in Review, Regulatory
Novartis AG (NYSE:NVS; SIX:NOVN), Basel, Switzerland Product: Mavoglurant (AFQ056) Business: Neurology The European Commission granted Orphan Drug designation to Novartis’ mavoglurant to treat fragile X syndrome. The …

ماووگلوران (به انگلیسی: Mavoglurant)‏ یک ترکیب شیمیایی با شناسه پاب‌کم ۹۹۲۶۸۳۲ است.

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4 Comments

  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 AFRICURE PHARMA as ADVISOR, earlier assignment was with GLENMARK LIFE SCIENCES LTD, as CONSUlTANT, Retired from GLENMARK in Jan2022 Research Centre as Principal Scientist, Process Research (bulk actives) at Mahape, Navi Mumbai, India. Total Industry exp 32 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, 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 32 PLUS year tenure till date Feb 2023, 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 100 million plus hits on Google, 2.5 lakh plus connections on all networking sites, 100 Lakh plus views on dozen plus blogs, 227 countries, 7 continents, 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 38 lakh plus views on New Drug Approvals Blog in 227 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 He has total of 32 International and Indian awards

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