syounika/jdpt/reguratori/check/pdf/16/15_laroni.pdf · 5—2. PK Emil D. Kakkis et al. The New...

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Transcript of syounika/jdpt/reguratori/check/pdf/16/15_laroni.pdf · 5—2. PK Emil D. Kakkis et al. The New...

Page 1: syounika/jdpt/reguratori/check/pdf/16/15_laroni.pdf · 5—2. PK Emil D. Kakkis et al. The New England J of Med. vol.334 No.3 p 182-188, 2001 10 1 1 125,000U/kg 52
Page 2: syounika/jdpt/reguratori/check/pdf/16/15_laroni.pdf · 5—2. PK Emil D. Kakkis et al. The New England J of Med. vol.334 No.3 p 182-188, 2001 10 1 1 125,000U/kg 52
Page 3: syounika/jdpt/reguratori/check/pdf/16/15_laroni.pdf · 5—2. PK Emil D. Kakkis et al. The New England J of Med. vol.334 No.3 p 182-188, 2001 10 1 1 125,000U/kg 52
Page 4: syounika/jdpt/reguratori/check/pdf/16/15_laroni.pdf · 5—2. PK Emil D. Kakkis et al. The New England J of Med. vol.334 No.3 p 182-188, 2001 10 1 1 125,000U/kg 52

Improving the Clinical Development Process Disclaimer · Disclaimer: Presentation slides from the Rare Disease Workshop Series are posted by the Kakkis EveryLife Foundation, for educational

Improving the Clinical Development Process Disclaimer · Disclaimer: Presentation slides from the Rare Disease Workshop Series are posted by the Kakkis EveryLife Foundation, for educational

syounika/jdpt/reguratori/check/pdf/17/16... · 8-1 03-3353-8112 37308 FAX 03-5379-1440 E—mai I mosawa@ped. twmu. ac. jp FAX E—mai I

syounika/jdpt/reguratori/check/pdf/17/16... · 8-1 03-3353-8112 37308 FAX 03-5379-1440 E—mai I mosawa@ped. twmu. ac. jp FAX E—mai I

C URE T HE P ROCESS 2: Improving the Development Process for Rare Disease Treatments Emil D. Kakkis, M.D., Ph.D. President and Founder October 29, 2014.

C URE T HE P ROCESS 2: Improving the Development Process for Rare Disease Treatments Emil D. Kakkis, M.D., Ph.D. President and Founder October 29, 2014.

香川大学 医学部・医学系研究科syounika/jdpt/reguratori/check/...Cecil Essentials of Medicine, 6th edition Nelson Textbook of Pediatrics, 17th edition Harrison' s Internal

香川大学 医学部・医学系研究科syounika/jdpt/reguratori/check/...Cecil Essentials of Medicine, 6th edition Nelson Textbook of Pediatrics, 17th edition Harrison' s Internal

1. a. b. C. d. 2. a. b. 3. a. b. c. d. 5. Cochrane …syounika/jdpt/reguratori/check/...Cochrane Review Care of the high-risk neonate, 5 296, 2001, WB Saunders Company Textbook Of

1. a. b. C. d. 2. a. b. 3. a. b. c. d. 5. Cochrane …syounika/jdpt/reguratori/check/...Cochrane Review Care of the high-risk neonate, 5 296, 2001, WB Saunders Company Textbook Of

syounika/jdpt/reguratori/check/...b) (2,000mg/kg/H) (1)Bile acids for viral hepatitis Chen W, Liu J, Gluud C. Cochrane Hepat0-Biliary Group, Copenhagen Trial Unit, Denmark Cochrane

syounika/jdpt/reguratori/check/...b) (2,000mg/kg/H) (1)Bile acids for viral hepatitis Chen W, Liu J, Gluud C. Cochrane Hepat0-Biliary Group, Copenhagen Trial Unit, Denmark Cochrane

syounika/jdpt/reguratori/check/pdf/14/27_soru1.pdf · MPT Often, 68.50/0 sometimes Mendaza MPT MPT 7 MPT MPT 7 MPT 7 23 46 h 12 MPT 1-2 12 MPT 24 33 U, y 2001 D L. 4 . 10 1959 Bib,

syounika/jdpt/reguratori/check/pdf/14/27_soru1.pdf · MPT Often, 68.50/0 sometimes Mendaza MPT MPT 7 MPT MPT 7 MPT 7 23 46 h 12 MPT 1-2 12 MPT 24 33 U, y 2001 D L. 4 . 10 1959 Bib,

香川大学 医学部・医学系研究科syounika/jdpt/reguratori/check/...3. WII l. Neonatology: 4thed, Avery GB et al., Lippincott, Philadelphia, 1994, p411, p1291, p1441. 2. Nelson

香川大学 医学部・医学系研究科syounika/jdpt/reguratori/check/...3. WII l. Neonatology: 4thed, Avery GB et al., Lippincott, Philadelphia, 1994, p411, p1291, p1441. 2. Nelson

syounika/jdpt/reguratori/check/...care Medicine, 169(7), A89—A89, = F D 7— —d 2004 BE-I.2crn, (-1.9cm. U @Szefier S : New England Journal of Medicine,343, 1054 — 1063, 2000

syounika/jdpt/reguratori/check/...care Medicine, 169(7), A89—A89, = F D 7— —d 2004 BE-I.2crn, (-1.9cm. U @Szefier S : New England Journal of Medicine,343, 1054 — 1063, 2000

Welcome to Workshop #5: Accelerated Approval (AA) in Rare Diseases: Review of a White Paper Proposal Emil D. Kakkis, M.D., Ph.D. President and Founder.

Welcome to Workshop #5: Accelerated Approval (AA) in Rare Diseases: Review of a White Paper Proposal Emil D. Kakkis, M.D., Ph.D. President and Founder.

Emil D. Kakkis, M.D., Ph.D. President and Founder EveryLife Foundation for Rare Diseases

Emil D. Kakkis, M.D., Ph.D. President and Founder EveryLife Foundation for Rare Diseases

syounika/jdpt/reguratori/check/pdf/15/9_ri... · Zuddas,A. et al, J.Child Adoles.Psychopharmacol. : Mcdougle,C.J. et al, J .Am. Acad. Child Adolesc. Psychiatry, 1997 n=34Ð1 / (6-62

syounika/jdpt/reguratori/check/pdf/15/9_ri... · Zuddas,A. et al, J.Child Adoles.Psychopharmacol. : Mcdougle,C.J. et al, J .Am. Acad. Child Adolesc. Psychiatry, 1997 n=34Ð1 / (6-62

Improving the Process of Rare Disease Treatment Development Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President, Ultragenyx Pharmaceutical,

Improving the Process of Rare Disease Treatment Development Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President, Ultragenyx Pharmaceutical,

syounika/jdpt/reguratori/check/pdf/14/30... · (AECB) and Bacterial Pneumonia (BP). 42nd ICAAC, 345, sep. 2002, san Diego, USA ... Population Pharmacokinetics (PPK) of Ceftizoxime.Alapivoxil

syounika/jdpt/reguratori/check/pdf/14/30... · (AECB) and Bacterial Pneumonia (BP). 42nd ICAAC, 345, sep. 2002, san Diego, USA ... Population Pharmacokinetics (PPK) of Ceftizoxime.Alapivoxil