Table of Contents List of Tables List of Figures Introduction Global Markets Direct Report Coverage Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Overview Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Therapeutics Development Products under Development by Stage of Development Products under Development by Therapy Area Products under Development by Indication Products under Development by Companies Products under Development by Universities/Institutes Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Therapeutics Assessment Assessment by Mechanism of Action Assessment by Route of Administration Assessment by Molecule Type Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Companies Involved in Therapeutics Development AveXis Inc Biogen Inc Exicure Inc F. Hoffmann-La Roche Ltd Ionis Pharmaceuticals Inc Novartis AG Sarepta Therapeutics Inc Spotlight Innovation Inc Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Drug Profiles ALB-111 - Drug Profile Product Description Mechanism Of Action R&D Progress Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Antisense Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile Product Description Mechanism Of Action R&D Progress BIIB-089 - Drug Profile Product Description Mechanism Of Action R&D Progress branaplam - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile Product Description Mechanism Of Action R&D Progress IONISENAC-2.5Rx - Drug Profile Product Description Mechanism Of Action R&D Progress LDN-5178 - Drug Profile Product Description Mechanism Of Action R&D Progress Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile Product Description Mechanism Of Action R&D Progress onasemnogene abeparvovec - Drug Profile Product Description Mechanism Of Action R&D Progress PMO-25 - Drug Profile Product Description Mechanism Of Action R&D Progress risdiplam - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules 2 to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules for Spinal Muscular Atrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile Product Description Mechanism Of Action R&D Progress STL-182 - Drug Profile Product Description Mechanism Of Action R&D Progress Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Dormant Products Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Discontinued Products Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Product Development Milestones Featured News & Press Releases Apr 03, 2019: ICER Issues Final Report on Zolgensma, provides Policy recommendations related to Pricing and Coverage of treatments for Spinal Muscular Atrophy Apr 01, 2019: AveXis expands world-leading gene therapy manufacturing capacity with purchase of advanced biologics therapy manufacturing campus in Longmont, Colorado Mar 27, 2019: Chugai receives Orphan Drug Designation for Risdiplam in Spinal Muscular Atrophy Feb 18, 2019: Gene Therapy Firm AveXis to expand in Durham County adding 200 jobs and $60 Million in investment Feb 01, 2019: First phase 3 gene therapy trial in spinal muscular atrophy at The John Walton Muscular Dystrophy Centre Jan 08, 2019: Genentech, Roche and PTC Therapeutics provide update on Regulatory Filing for Risdiplam Dec 19, 2018: Roche gets PRIME designation for SMA drug in Europe Dec 03, 2018: Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1 Nov 05, 2018: Novartis provides update on AVXS101 Oct 03, 2018: Roche announces new data for Risdiplam in spinal muscular atrophy(SMA) at the World Muscle Society Congress Oct 03, 2018: Risdiplam Demonstrates Preliminary Evidence of Clinical Benefit in Type 1, 2, & 3 Spinal Muscular Atrophy Patients Jul 24, 2018: Spotlight Innovation spinal muscular atrophy research collaborator professor Kevin Hodgetts receives $300,000 grant from Cure SMA Jun 16, 2018: Updated Preliminary Data from SMA FIREFISH Program in Type 1 Babies Presented at the CureSMA Conference Jun 14, 2018: Exicure to Present Data at the Cure Spinal Muscular Atrophy Annual Conference in Dallas May 08, 2018: AveXis Issues Community Statement on the SPR1NT Trial Appendix Methodology Coverage Secondary Research Primary Research Expert Panel Validation Contact Us Disclaimer

List of Tables Number of Products under Development by Stage of Development, H1 2019 Number of Products under Development by Therapy Areas, H1 2019 Number of Products under Development by Indication, H1 2019 Number of Products under Development by Companies, H1 2019 Products under Development by Companies, H1 2019 Number of Products under Investigation by Universities/Institutes, H1 2019 Products under Investigation by Universities/Institutes, H1 2019 Number of Products by Stage and Mechanism of Actions, H1 2019 Number of Products by Stage and Route of Administration, H1 2019 Number of Products by Stage and Molecule Type, H1 2019 Pipeline by AveXis Inc, H1 2019 Pipeline by Biogen Inc, H1 2019 Pipeline by Exicure Inc, H1 2019 Pipeline by F. Hoffmann-La Roche Ltd, H1 2019 Pipeline by Ionis Pharmaceuticals Inc, H1 2019 Pipeline by Novartis AG, H1 2019 Pipeline by Sarepta Therapeutics Inc, H1 2019 Pipeline by Spotlight Innovation Inc, H1 2019 Dormant Projects, H1 2019 Discontinued Products, H1 2019List of Figures Number of Products under Development by Stage of Development, H1 2019 Number of Products under Development by Therapy Areas, H1 2019 Number of Products under Development by Top 10 Indications, H1 2019 Number of Products by Stage and Mechanism of Actions, H1 2019 Number of Products by Routes of Administration, H1 2019 Number of Products by Stage and Routes of Administration, H1 2019 Number of Products by Molecule Types, H1 2019 Number of Products by Stage and Molecule Types, H1 2019

AveXis Inc Biogen Inc Exicure Inc F. Hoffmann-La Roche Ltd Ionis Pharmaceuticals Inc Novartis AG Sarepta Therapeutics Inc Spotlight Innovation Inc