Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Spil Muscular Atrophy (SMA) - Overview
Spil Muscular Atrophy (SMA) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Spil Muscular Atrophy (SMA) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Spil Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development
Alcyone Therapeutics Inc
AlphaCognition Inc
Amniotics AB
Anima Biotech Inc
Annji Pharmaceutical Co Ltd
Apteeus SAS
ARMGO Pharma Inc
Beijing GeneCradle Technology Co Ltd
Biocad
Biogen Inc
Biohaven Pharmaceutical Holding Company Ltd
Bioleaders Corp
BioMarin Pharmaceutical Inc
Biophytis SA
Cell Tech Pharmed Co
Chugai Pharmaceutical Co Ltd
Enzer Biosciences LLC
Exegenesis Bio Inc
F. Hoffmann-La Roche Ltd
Genentech USA Inc
GNT Pharma Co Ltd
Huida (Shanghai) Biotechnology Co Ltd
Imago Pharmaceuticals Inc
Myocea Inc
Neurotune AG
NMD Pharma AS
Novartis AG
Novartis Gene Therapies
Oncterl Therapeutics
Ono Pharmaceutical Co Ltd
PTC Therapeutics Inc
Ractigen Therapeutics Inc
Rebor Biosciences Inc
Sarepta Therapeutics Inc
Scholar Rock Inc
Shift Pharmaceuticals
Spotlight Innovation Inc
Suzhou Gessist Therapeutics Co Ltd
Voyager Therapeutics Inc
Vybion Inc
Spil Muscular Atrophy (SMA) - Drug Profiles
AAD-2004 - Drug Profile
Product Description
Mechanism Of Action
ACTX-401 - Drug Profile
Product Description
Mechanism Of Action
History of Events
ALB-111 - Drug Profile
Product Description
Mechanism Of Action
ALPHA-0602 - Drug Profile
Product Description
Mechanism Of Action
ALZ-002 - Drug Profile
Product Description
Mechanism Of Action
History of Events
amifampridine phosphate - Drug Profile
Product Description
Mechanism Of Action
History of Events
ANB-4 - Drug Profile
Product Description
Mechanism Of Action
Antisense Oligonucleotide to Activate SMN2 for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Antisense Oligonucleotides for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
apitegromab - Drug Profile
Product Description
Mechanism Of Action
History of Events
ARM-210 - Drug Profile
Product Description
Mechanism Of Action
History of Events
BIIB-089 - Drug Profile
Product Description
Mechanism Of Action
History of Events
BIIB-110 - Drug Profile
Product Description
Mechanism Of Action
History of Events
BIIB-115 - Drug Profile
Product Description
Mechanism Of Action
History of Events
BIO-101 - Drug Profile
Product Description
Mechanism Of Action
History of Events
BLSM-22 - Drug Profile
Product Description
Mechanism Of Action
braplam hydrochloride - Drug Profile
Product Description
Mechanism Of Action
History of Events
CogniStem - Drug Profile
Product Description
Mechanism Of Action
Drugs for Neuromuscular Diseases and Spil Muscular Atrophy (SMA) - Drug Profile
Product Description
Mechanism Of Action
History of Events
E-1V111 - Drug Profile
Product Description
Mechanism Of Action
History of Events
ENZ-001 - Drug Profile
Product Description
Mechanism Of Action
History of Events
GC-101 - Drug Profile
Product Description
Mechanism Of Action
GEN-8010 - Drug Profile
Product Description
Mechanism Of Action
Gene Therapies to Activate SMN1 for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate SMN1 for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate SYT13 for Amyotrophic Lateral Sclerosis and Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
GTP-201 - Drug Profile
Product Description
Mechanism Of Action
History of Events
GTx-505 - Drug Profile
Product Description
Mechanism Of Action
History of Events
HG-001 - Drug Profile
Product Description
Mechanism Of Action
INT-41 - Drug Profile
Product Description
Mechanism Of Action
History of Events
maresin-1 - Drug Profile
Product Description
Mechanism Of Action
Monoclol Antibody to Inhibit Myostatin for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
History of Events
NMDP-02 - Drug Profile
Product Description
Mechanism Of Action
NT-1654 - Drug Profile
Product Description
Mechanism Of Action
osemnogene abeparvovec - Drug Profile
Product Description
Mechanism Of Action
History of Events
ONCT-534 - Drug Profile
Product Description
Mechanism Of Action
History of Events
PMO-25 - Drug Profile
Product Description
Mechanism Of Action
History of Events
RAG-06 - Drug Profile
Product Description
Mechanism Of Action
Resagen - Drug Profile
Product Description
Mechanism Of Action
History of Events
RG-6237 - Drug Profile
Product Description
Mechanism Of Action
History of Events
risdiplam - Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecule for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecule to Inhibit GSK-3 for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Small Molecules 2 to Activate SMN2 for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Small Molecules for Central Nervous System Disorders - Drug Profile
Product Description
Mechanism Of Action
Small Molecules for Kennedy’s Disease - Drug Profile
Product Description
Mechanism Of Action
Small Molecules for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Small Molecules for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecules for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Small Molecules to Activate SMN2 for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Small Molecules to Antagonize AR for Spil and Bulbar Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
History of Events
STL-182 - Drug Profile
Product Description
Mechanism Of Action
History of Events
talditercept alfa - Drug Profile
Product Description
Mechanism Of Action
History of Events
TEC-1 - Drug Profile
Product Description
Mechanism Of Action
TEE-375 - Drug Profile
Product Description
Mechanism Of Action
TEE-703 - Drug Profile
Product Description
Mechanism Of Action
timrepigene emparvovec - Drug Profile
Product Description
Mechanism Of Action
History of Events
Vartocell - Drug Profile
Product Description
Mechanism Of Action
Spil Muscular Atrophy (SMA) - Dormant Projects
Spil Muscular Atrophy (SMA) - Discontinued Products
Spil Muscular Atrophy (SMA) - Product Development Milestones
Featured News & Press Releases
Mar 22, 2022: Scholar Rock presents data alysis of multiple efficacy endpoints from the apitegromab TOPAZ phase 2 trial at the American Academy of Neurology 2022 Annual Meeting
Mar 16, 2022: New data for Roche?s Evrysdi (risdiplam) demonstrate long-term efficacy and safety in a broad population of people with spil muscular atrophy (SMA)
Mar 16, 2022: New data for Genentech?s Evrysdi (risdiplam) demonstrate long-term efficacy and safety in a broad population of people with Spil Muscular Atrophy (SMA)
Mar 13, 2022: Scholar Rock to present data from TOPAZ Ambulatory Cohort Alysis at the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
Mar 13, 2022: Novartis data again demonstrate age-appropriate development when Zolgensma is used presymptomatically, and post-hoc data reveal SMA Type 1 patients could speak, swallow and maintain airway protection
Mar 08, 2022: Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio
Feb 03, 2022: Roche Cada and the pan-Cadian Pharmaceutical Alliance (pCPA) successfully complete negotiations for EVRYSDI (risdiplam) for the treatment of adults and children with spil muscular atrophy (SMA)
Jan 31, 2022: Cost-effectiveness alysis temporarily halted for Zolgensma to collect long-term data
Jan 25, 2022: Roche’s Evrysdi (risdiplam) granted FDA priority review for treatment of pre-symptomatic babies under 2 months of age with spil muscular atrophy (SMA)
Nov 30, 2021: Scholar Rock announces design of phase 3 SAPPHIRE clinical trial evaluating apitegromab in non-ambulatory patients with Type 2 and Type 3 spil muscular atrophy (SMA)
Nov 19, 2021: NICE draft guidance recommends new treatment for spil muscular atrophy as part of a maged access agreement
Oct 19, 2021: Novartis applauds Quebec, the first province to list Zolgensma for the treatment of pediatric patients with spil muscular atrophy (SMA)
Oct 13, 2021: Novartis and the pan-Cadian Pharmaceutical Alliance (pCPA) complete negotiations for Zolgensma for the treatment of pediatric patients with spil muscular atrophy (SMA)
Oct 03, 2021: Scholar Rock to present Apitegromab TOPAZ phase 2 pharmacologic data at the 2021 World Congress of Neurology
Sep 30, 2021: Scholar Rock presents exploratory responder alysis on efficacy data from the apitegromab TOPAZ phase 2 trial at the Child Neurology Society Annual Meeting
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures

List of Figures
Number of Products under Development for Spinal Muscular Atrophy (SMA), 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Number of Products by Top 10 Targets, 2022
Number of Products by Stage and Top 10 Targets, 2022
Number of Products by Top 10 Mechanism of Actions, 2022
Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Top 10 Molecule Types, 2022
Number of Products by Stage and Top 10 Molecule Types, 2022

List of Tables

List of Tables
Number of Products under Development for Spinal Muscular Atrophy (SMA), 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Companies, 2022 (Contd..1)
Number of Products under Development by Universities/Institutes, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Products under Development by Companies, 2022 (Contd..2)
Products under Development by Companies, 2022 (Contd..3)
Products under Development by Universities/Institutes, 2022
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Alcyone Therapeutics Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by AlphaCognition Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Amniotics AB, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Anima Biotech Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Annji Pharmaceutical Co Ltd, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Apteeus SAS, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by ARMGO Pharma Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Beijing GeneCradle Technology Co Ltd, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Biocad, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Biogen Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Biohaven Pharmaceutical Holding Company Ltd, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Bioleaders Corp, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by BioMarin Pharmaceutical Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Biophytis SA, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Cell Tech Pharmed Co, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Chugai Pharmaceutical Co Ltd, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Enzerna Biosciences LLC, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Exegenesis Bio Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Genentech USA Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by GNT Pharma Co Ltd, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Huida (Shanghai) Biotechnology Co Ltd, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Imago Pharmaceuticals Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Myocea Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by NMD Pharma AS, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Novartis Gene Therapies, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Oncternal Therapeutics, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Ono Pharmaceutical Co Ltd, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by PTC Therapeutics Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Ractigen Therapeutics Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Reborna Biosciences Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Scholar Rock Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Shift Pharmaceuticals, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Spotlight Innovation Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Suzhou GenAssist Therapeutics Co Ltd, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Voyager Therapeutics Inc, 2022
Spinal Muscular Atrophy (SMA) - Pipeline by Vybion Inc, 2022
Spinal Muscular Atrophy (SMA) - Dormant Projects, 2022
Spinal Muscular Atrophy (SMA) - Dormant Projects, 2022 (Contd..1)
Spinal Muscular Atrophy (SMA) - Dormant Projects, 2022 (Contd..2)
Spinal Muscular Atrophy (SMA) - Discontinued Products, 2022