SG11201804915RA - Methods for treating huntington's disease - Google Patents

Methods for treating huntington's disease

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Publication number
SG11201804915RA
SG11201804915RA SG11201804915RA SG11201804915RA SG11201804915RA SG 11201804915R A SG11201804915R A SG 11201804915RA SG 11201804915R A SG11201804915R A SG 11201804915RA SG 11201804915R A SG11201804915R A SG 11201804915RA SG 11201804915R A SG11201804915R A SG 11201804915RA
Authority
SG
Singapore
Prior art keywords
international
disease
forms
compounds
methods
Prior art date
Application number
SG11201804915RA
Inventor
Suresh Babu
Anuradha Bhattacharyya
Seongwoo Hwang
Minakshi Jani
Young-Choon Moon
Nadiya Sydorenko
Original Assignee
Ptc Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority to US201562265652P priority Critical
Application filed by Ptc Therapeutics Inc filed Critical Ptc Therapeutics Inc
Priority to PCT/US2016/066042 priority patent/WO2017100726A1/en
Publication of SG11201804915RA publication Critical patent/SG11201804915RA/en

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    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/506Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
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    • A61K31/41Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
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    • G01N2800/2835Movement disorders, e.g. Parkinson, Huntington, Tourette

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property , Organization MD 1111101110101011111 HO 11111 011101011101110111 OH III 111110 ill OEN International Bureau .. .... ..Yejd (10) International Publication Number (43) International Publication Date ..... ...r .....1 WO 2017/100726 Al 15 June 2017(15.06.2017) WIPO I PCT (51) International Patent Classification: (81) Designated States (unless otherwise indicated, for every A61K 31/506 (2006.01) A61P 25/00 (2006.01) kind of national protection available): AE, AG, AL, AM, CO7D 405/12 (2006.01) AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, (21) International Application Number: DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, PCT/US2016/066042 HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, (22) International Filing Date: KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, 11 December 2016 (11.12.2016) MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, (25) Filing Language: English RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, (26) Publication Language: English TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (30) Priority Data: 62/265,652 10 December 2015 (10.12.2015) US (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, (71) Applicant: PTC THERAPEUTICS, INC. [US/US]; 100 GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, Corporate Court, South Plainfield, NJ 07080 (US). TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, (72) TJ, TM), Inventors: BABU, Suresh; 225 Concord Place, Penning- European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, ton, NJ 08534 (US). BHATTACHARYYA, Anuradha; EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, 115 Inverness Drive, Edison, NJ 08820 (US). HWANG, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, Seongwoo; 227 Suttons Lane, Edison, NJ 08817 (US). SM, GW, KM, ML, MR, NE, SN, TD, TG). TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, JANE Minakshi; 29 Kiva Street, Iselin, NJ 08830 (US). MOON, Young-choon; 11 Edgewood Drive, Belle Mead, Published: NJ 08502 (US). SYDORENKO, Nadiya; 37 Sycamore with Place, Princento, NJ 08540 (US). international search report (Art. 21(3)) (74) Agents: SCOLA, Daniel, A., Jr. et al.; Hoffmann & Bar- on, LLP, 6900 Jericho Turnpike, Syosset, NY 11791 (US). (54) Title: METHODS FOR TREATNG HUNTINGTON'S DISEASE W A 41111.1.1CP , \"s r .- X....... / N — N (I) ,tD ei IN © © ,-1 IN 1-1 (57) : The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using 0 such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present descrip - N tion relates to substituted monocyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and 0 methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
SG11201804915RA 2015-12-10 2016-12-11 Methods for treating huntington's disease SG11201804915RA (en)

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Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2018007022A (en) 2015-12-10 2018-11-12 Ptc Therapeutics Inc Methods for treatng huntington's disease.
WO2019005993A1 (en) * 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
GB2579747A (en) * 2017-08-04 2020-07-01 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
BR112020019373A2 (en) * 2018-03-27 2020-12-29 Ptc Therapeutics, Inc. COMPOUNDS FOR THE TREATMENT OF HUTINGTON'S DISEASE
WO2019199972A1 (en) * 2018-04-10 2019-10-17 Skyhawk Therapeutics, Inc. Compounds for the treatment of cancer
WO2020005882A1 (en) * 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
WO2020005873A1 (en) * 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating huntington's disease
AU2019294482A1 (en) * 2018-06-27 2021-01-21 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
WO2020163323A1 (en) * 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163375A1 (en) * 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163401A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163405A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163541A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163409A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163406A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163647A1 (en) * 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163544A1 (en) * 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020190793A1 (en) * 2019-03-15 2020-09-24 Skyhawk Therapeutics, Inc. Compositions and methods for correction of aberrant splicing
WO2020231977A1 (en) * 2019-05-13 2020-11-19 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
WO2020234715A1 (en) * 2019-05-17 2020-11-26 Novartis Ag Nlrp3 inflammasome inhibitors
WO2021014428A1 (en) 2019-07-25 2021-01-28 Novartis Ag Regulatable expression systems

Family Cites Families (97)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3558618A (en) 1968-04-01 1971-01-26 Dow Chemical Co Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones
GB1383409A (en) 1972-09-09 1974-02-12 Pfizer Ltd Derivatives of 2-amino- and 4-amino-quinazoline and pharmaceutical compositions containing them
US4122274A (en) 1977-05-25 1978-10-24 Bristol-Myers Company 3-Tetrazolo-5,6,7,8-substituted-pyrido[1,2-a]pyrimidin-4-ones
US4342870A (en) 1980-03-28 1982-08-03 Janssen Pharmaceutica N.V. Novel 3-(1-piperidinylalkyl)-4H-pyrido[1,2-a]pyrimidin-4-one derivatives
JPH0215550B2 (en) 1980-03-28 1990-04-12 Janssen Pharmaceutica Nv
US5089633A (en) 1987-04-28 1992-02-18 Georgia Tech Research Corporation Substituted isocoumarins
US5599816A (en) 1990-05-02 1997-02-04 Abbott Laboratories Quinolizinone type compounds
AU4231293A (en) 1992-05-13 1993-12-13 E.I. Du Pont De Nemours And Company Substituted pyrido(1,2-A)pyrimidinone derivatives as fungicides
AU6153096A (en) 1995-06-06 1996-12-24 Abbott Laboratories Quinolizinone type compounds
US5916916A (en) 1996-10-10 1999-06-29 Eli Lilly And Company 1-aryloxy-2-arylnaphthyl compounds, intermediates, compositions, and methods
US5869500A (en) 1996-12-13 1999-02-09 Hoffmann-La Roche Inc. Pyridone compounds useful in treating Alzheimer's disease
CA2269561C (en) 1998-04-22 2007-06-05 Dainippon Ink And Chemicals, Inc. Naphthalene derivative and liquid crystal composition comprising the same
AU5991699A (en) 1998-09-21 2000-04-10 Biochem Pharma Inc. Quinolizinones as integrin inhibitors
AT312820T (en) 1999-10-28 2005-12-15 Trine Pharmaceuticals Inc Pump inhibitors for the release of medicaments
CA2398163C (en) 2000-01-24 2011-02-22 Kinacia Pty Ltd. Therapeutic morpholino-substituted compounds
US6774134B2 (en) 2000-12-20 2004-08-10 Bristol-Myers Squibb Company Heterocyclic substituted 2-methyl-benzimidazole antiviral agents
KR20030061858A (en) 2000-12-21 2003-07-22 버텍스 파마슈티칼스 인코포레이티드 Pyrazole compounds useful as protein kinase inhibitors
CA2445697A1 (en) 2001-04-26 2002-11-07 Daiichi Pharmaceutical Co., Ltd. Drug efflux pump inhibitor
WO2003049739A1 (en) 2001-12-07 2003-06-19 Vertex Pharmaceuticals, Inc. Pyrimidine-based compounds useful as gsk-3 inhibitors
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
AT548354T (en) 2002-07-24 2012-03-15 Ptc Therapeutics Inc Ureido-substituted benzoic acid compounds and their use in nonsense suppression and treatment of illnesses
EP1785425B1 (en) 2002-09-30 2009-03-25 NeuroSearch A/S 1,4-Diazabicycloalkane derivatives, their preparation and use
US20070078144A1 (en) 2003-01-29 2007-04-05 Stockwell Brent R Agents for treating neurodegenerative diseases
KR20060069356A (en) 2003-06-20 2006-06-21 카이론 코포레이션 Pyridino[1,2-a]pyrimidin-4-one compounds as anticancer agents
GB0315494D0 (en) 2003-07-02 2003-08-06 Biofocus Plc Compounds which bind to the active site of protein kinase enzymes
US7309699B2 (en) 2003-12-22 2007-12-18 Abbott Laboratories 3-Quinuclidinyl amino-substituted biaryl derivatives
US7655657B2 (en) * 2003-12-22 2010-02-02 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US20050245531A1 (en) 2003-12-22 2005-11-03 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US8598193B2 (en) 2003-12-24 2013-12-03 Biota Scientific Management Pty Ltd. Polycyclic agents for the treatment of respiratory syncytial virus infections
SE0400184D0 (en) 2004-01-30 2004-01-30 Fyrkloevern Scandinavia Ab New therapeutic use
JP2007532570A (en) 2004-04-08 2007-11-15 ニューロジェン・コーポレーション Substituted cinnolin-4-ylamines
DE602005002562T2 (en) 2004-05-04 2008-01-31 Warner-Lambert Company Llc PYRROLYL SUBSTITUTED PYRIDOE2,3-DIPYRIMIDIN-7-ONE AND DERIVATIVES THEREOF AS THERAPEUTIC AGENTS
CA2595477A1 (en) 2005-01-21 2006-07-27 Janssen Pharmaceutica N.V. Novel heterocyclic benzo[c]chromene derivatives useful as modulators of the estrogen receptors
US8501739B2 (en) * 2005-07-04 2013-08-06 High Point Pharmaceuticals, Llc Medicaments
AR059339A1 (en) 2006-02-09 2008-03-26 Chugai Pharmaceutical Co Ltd Coumarin derivatives for cell proliferative disorders, pharmaceutical composition and therapeutic agent containing them
US8110681B2 (en) 2006-03-17 2012-02-07 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Compounds for the treatment of spinal muscular atrophy and other uses
WO2007130383A2 (en) * 2006-04-28 2007-11-15 Northwestern University Compositions and treatments using pyridazine compounds and secretases
US20080247964A1 (en) * 2006-05-08 2008-10-09 Yuelian Xu Substituted azaspiro derivatives
JP2009537599A (en) * 2006-05-23 2009-10-29 ノイロサーチ アクティーゼルスカブ Novel 8,10-diaza-bicyclo [4.3.1] decane derivatives and their medical use
WO2008013997A2 (en) 2006-07-27 2008-01-31 The Trustees Of Columbia University In The City Of New York Fluorescent substrates for monoamine transporters as optical false neurotransmitters
EP2079734A1 (en) 2006-10-25 2009-07-22 NeuroSearch A/S Oxadiazole and thiadiazole compounds and their use as nicotinic acetylcholine receptor modulators
US8314119B2 (en) * 2006-11-06 2012-11-20 Abbvie Inc. Azaadamantane derivatives and methods of use
EP2114903B1 (en) 2006-12-22 2011-01-26 Avexa Limited Bicyclic pyrimidinones and uses thereof
US20080171792A1 (en) 2006-12-28 2008-07-17 Jobdevairakkam Christopher New Use of highly concentrated formulations of 4-phenylbutyrate for treatment of certain disorders
FR2914188B1 (en) 2007-03-28 2012-06-22 Trophos New cholest-4-en-3-one oxime composition
EP2014656A3 (en) * 2007-06-11 2011-08-24 High Point Pharmaceuticals, LLC New heteocyclic h3 antagonists
WO2009042907A1 (en) 2007-09-27 2009-04-02 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isoindoline compounds for the treatment of spinal muscular atrophy and other uses
US20090264433A1 (en) 2008-04-21 2009-10-22 Institute For Oneworld Health Compounds, Compositions and Methods Comprising Triazine Derivatives
US8633019B2 (en) 2008-05-27 2014-01-21 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
CN102159208B (en) 2008-06-20 2014-05-28 罗塔药品股份有限公司 6-1h-imidazo-quinazoline and quinolines derivatives, new mao inhibitors and imidazoline receptor ligands
EP2138493A1 (en) 2008-06-26 2009-12-30 Sanofi-Aventis Substituted pyrimidone derivatives
PL2212324T3 (en) 2008-07-02 2014-11-28 Avexa Ltd Compounds having antiviral properties
US8932818B2 (en) 2008-08-13 2015-01-13 Ptc Therapeutics, Inc. Screening assays for compounds that modulate programmed ribosomal frameshifting
GB2465405A (en) 2008-11-10 2010-05-19 Univ Basel Triazine, pyrimidine and pyridine analogues and their use in therapy
US8063032B2 (en) 2009-02-11 2011-11-22 Sunovion Pharmaceuticals Inc. Histamine H3 inverse agonists and antagonists and methods of use thereof
RU2015110025A (en) * 2009-06-19 2015-08-20 Эббви Инк. Diazagomoadamantane derivatives and ways of their application
WO2011050245A1 (en) 2009-10-23 2011-04-28 Thomas Bannister Bicyclic heteroaryls as kinase inhibitors
EP2501231B1 (en) 2009-11-20 2016-12-21 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide m1 receptor positive allosteric modulators
AU2011206761A1 (en) 2010-01-13 2012-07-12 Institut National De La Sante Et De La Recherche Medicale (Inserm) Anti - infective pyrido (1,2 -a) pyrimidines
CA2800509A1 (en) 2010-05-24 2011-12-01 Presidio Pharmaceuticals, Inc. Inhibitors of hcv ns5a
US20120008349A1 (en) * 2010-07-12 2012-01-12 Scharf Mesa P Power inverter systems with high-accuracy reference signal generation and associated methods of control
WO2012075393A2 (en) * 2010-12-02 2012-06-07 President And Fellows Of Harvard College Activators of proteasomal degradation and uses thereof
US8703763B2 (en) 2011-03-02 2014-04-22 Hoffmann-La Roche Inc. Bridged piperidine derivatives
US8871756B2 (en) 2011-08-11 2014-10-28 Hoffmann-La Roche Inc. Compounds for the treatment and prophylaxis of Respiratory Syncytial Virus disease
WO2013059606A1 (en) 2011-10-21 2013-04-25 Tufts Medical Center, Inc. Compounds and methods for the treatment of muscular disease, and related screening methods
GB201119538D0 (en) 2011-11-10 2011-12-21 Viral Ltd Pharmaceutical compounds
BR112014012815A8 (en) 2011-11-28 2017-06-20 Novartis Ag trifluoromethyl oxadiazole derivatives and their use in the treatment of disease
KR102057351B1 (en) 2011-12-30 2019-12-18 피티씨 테라퓨틱스, 인크. Compounds for treating spinal muscular atrophy
US9399649B2 (en) 2012-01-26 2016-07-26 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
KR20200093066A (en) 2012-02-10 2020-08-04 피티씨 테라퓨틱스, 인크. Compounds for treating spinal muscular atrophy
CN104302181B (en) 2012-03-01 2017-09-15 Ptc医疗公司 Compound for treating Duchenne-Arandisease
CN104470909B (en) 2012-03-23 2018-04-24 Ptc医疗公司 For treating the compound of Duchenne-Arandisease
US8980934B2 (en) * 2012-06-22 2015-03-17 University Health Network Kinase inhibitors and method of treating cancer with same
AU2013289938A1 (en) 2012-07-13 2015-01-29 Indiana University Research & Technology Corporation Compounds for treatment of spinal muscular atrophy
US8729263B2 (en) 2012-08-13 2014-05-20 Novartis Ag 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
CA2910508A1 (en) 2013-05-14 2014-11-20 Song Feng Aza-oxo-indoles for the treatment and prophylaxis of respiratory syncytial virus infection
KR20160022312A (en) 2013-06-25 2016-02-29 에프. 호프만-라 로슈 아게 Compounds for treating spinal muscular atrophy
KR20160037199A (en) 2013-07-31 2016-04-05 노파르티스 아게 1,4-disubstituted pyridazine derivatives and their use for treating smn-deficiency-related conditions
WO2015024876A2 (en) 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
WO2015095449A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount rna transcripts
WO2015095446A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
WO2015105657A1 (en) 2013-12-19 2015-07-16 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
AR099134A1 (en) 2014-01-24 2016-06-29 F Hoffmann-La Roche Ag PROCEDURE FOR THE PREPARATION OF N - [(3-AMINOOXETAN-3-IL) METHYL] -2- (1,1-DIOXO-3,5-DIHIDRO-1,4-BENZOTIAZEPÍN-4-IL) -6-METIL- QUINAZOLÍN-4-AMINA
WO2015136947A1 (en) 2014-03-14 2015-09-17 Raqualia Pharma Inc. Azaspiro derivatives as trpm8 antagonists
PT3143025T (en) 2014-05-15 2019-12-03 Hoffmann La Roche Compounds for treating spinal muscular atrophy
EP3160967B1 (en) 2014-06-25 2019-05-08 F. Hoffmann-La Roche AG N-(imidazo[1,2-a]pyrazin-6-yl)-benzamide compounds for treating spinal muscular atrophy
US9920032B2 (en) 2015-10-02 2018-03-20 Incyte Corporation Heterocyclic compounds useful as pim kinase inhibitors
AU2016351919B2 (en) 2015-11-12 2020-11-12 F. Hoffmann-La Roche Ag Compounds for treating amyotrophic lateral sclerosis
EP3373972A1 (en) 2015-11-12 2018-09-19 H. Hoffnabb-La Roche Ag Compositions for treating spinal muscular atrophy
MX2018007022A (en) 2015-12-10 2018-11-12 Ptc Therapeutics Inc Methods for treatng huntington's disease.
AR108325A1 (en) 2016-04-27 2018-08-08 Samumed Llc ISOQUINOLIN-3-IL CARBOXAMIDS AND PREPARATION AND USE OF THE SAME
AU2018282154A1 (en) 2017-06-05 2020-01-02 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
WO2019005980A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
WO2019005993A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
GB2579747A (en) 2017-08-04 2020-07-01 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
BR112020019373A2 (en) 2018-03-27 2020-12-29 Ptc Therapeutics, Inc. COMPOUNDS FOR THE TREATMENT OF HUTINGTON'S DISEASE

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