Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study.

Saved in:
Bibliographic Details
Title: Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study.
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
Description
ISSN:1526-632X
DOI:10.1212/WNL.0000000000209151