Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study.
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| Title: | Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study. |
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| 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: | Neurology [Neurology] 2024 Mar 12; Vol. 102 (5), pp. e209151. Date of Electronic Publication: 2024 Feb 08. |
| Publication Type: | Randomized Controlled Trial; Clinical Trial, Phase III; Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | 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 |
| Database: | MEDLINE Ultimate |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 38330285 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Crawford+TO%22">Crawford TO</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Darras+BT%22">Darras BT</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Day+JW%22">Day JW</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Dunaway+Young+S%22">Dunaway Young S</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Duong+T%22">Duong T</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Nelson+LL%22">Nelson LL</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Barrett+D%22">Barrett D</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Song+G%22">Song G</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Bilic+S%22">Bilic S</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Cote+S%22">Cote S</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Sadanowicz+M%22">Sadanowicz M</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Iarrobino+R%22">Iarrobino R</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Xu+TJ%22">Xu TJ</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22O'Neil+J%22">O'Neil J</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Rossello+J%22">Rossello J</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Place+A%22">Place A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Kertesz+N%22">Kertesz N</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Nomikos+G%22">Nomikos G</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Chyung+Y%22">Chyung Y</searchLink>; 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. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%220401060%22">Neurology</searchLink> [Neurology] 2024 Mar 12; Vol. 102 (5), pp. e209151. <i>Date of Electronic Publication: </i>2024 Feb 08. – Name: TypePub Label: Publication Type Group: TypPub Data: Randomized Controlled Trial; Clinical Trial, Phase III; Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Lippincott+Williams+%26+Wilkins%22">Lippincott Williams & Wilkins </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>0401060 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1526-632X (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2200283878%22">00283878 </searchLink><i>NLM ISO Abbreviation: </i>Neurology <i>Subsets: </i>MEDLINE |
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| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1212/WNL.0000000000209151 Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: e209151 Titles: – TitleFull: Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Crawford TO – PersonEntity: Name: NameFull: Darras BT – PersonEntity: Name: NameFull: Day JW – PersonEntity: Name: NameFull: Dunaway Young S – PersonEntity: Name: NameFull: Duong T – PersonEntity: Name: NameFull: Nelson LL – PersonEntity: Name: NameFull: Barrett D – PersonEntity: Name: NameFull: Song G – PersonEntity: Name: NameFull: Bilic S – PersonEntity: Name: NameFull: Cote S – PersonEntity: Name: NameFull: Sadanowicz M – PersonEntity: Name: NameFull: Iarrobino R – PersonEntity: Name: NameFull: Xu TJ – PersonEntity: Name: NameFull: O'Neil J – PersonEntity: Name: NameFull: Rossello J – PersonEntity: Name: NameFull: Place A – PersonEntity: Name: NameFull: Kertesz N – PersonEntity: Name: NameFull: Nomikos G – PersonEntity: Name: NameFull: Chyung Y IsPartOfRelationships: – BibEntity: Dates: – D: 12 M: 03 Text: 2024 Mar 12 Type: published Y: 2024 Identifiers: – Type: issn-electronic Value: 1526-632X Numbering: – Type: volume Value: 102 – Type: issue Value: 5 Titles: – TitleFull: Neurology Type: main |
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