요약정보
Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study.
- Resource Type
- Academic Journal
- Authors
- Crawford TO; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Darras BT; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Day JW; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Dunaway Young S; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Duong T; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Nelson LL; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Barrett D; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Song G; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Bilic S; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Cote S; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Sadanowicz M; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Iarrobino R; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Xu TJ; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; O'Neil J; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Rossello J; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Place A; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Kertesz N; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Nomikos G; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.; Chyung Y; From the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.
- Source
- Publisher:
Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0401060 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1526-632X (Electronic) Linking ISSN:00283878 NLM ISO Abbreviation: Neurology Subsets: MEDLINE - Subject
- Language
- English