Spinal muscular atrophy

Emerging Therapeutics for Rare Neuromuscular Diseases: Pipeline Analysis

Retrieved on: 
Thursday, December 1, 2022
DN, Myasthenia gravis, MG, Cramp, Muscle weakness, RNA, Disorder, Duchenne muscular dystrophy, Therapy, Golodirsen, Patient, Motor neuron disease, Eteplirsen, Neuromuscular Disorders, US, Patent, Muscle, Amyotrophic lateral sclerosis, NMD, Spinal muscular atrophy, DMD, ALS, Diabetic neuropathy, NMDS, SMA, Quality of life, Myopathy, CIDP, US FDA, Degenerative disease, Dietary supplement, Pharmaceutical industry, Health insurance

Muscle weakness, cramps, and impaired musculoskeletal functions that occur due to rare defects in nerve and muscle cells characterize neuromuscular disorders (NMDs).

Key Points: 
  • Muscle weakness, cramps, and impaired musculoskeletal functions that occur due to rare defects in nerve and muscle cells characterize neuromuscular disorders (NMDs).
  • The disorders are largely classified as motor neuron diseases, hereditary ataxias, peripheral nerve disorders, neuromuscular junction transmission disorders, and myopathies.
  • Frost & Sullivans research, Emerging Therapeutics for Rare Neuromuscular Diseases: A Pipeline Analysis explores emerging biologics across these six NMDs.Key biologics emerging for these rare NMDs include stem cell therapies, other cell-based treatments, gene therapies, RNA therapeutics, immunotherapies, and other protein/peptide-based treatments.
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Sage Therapeutics and Biogen to Host Investor Webcast on December 6, 2022 to Discuss Potential Commercialization Plans for Zuranolone

Retrieved on: 
Tuesday, November 22, 2022
Biotechnology, Health, Science, Pharmaceutical, Research, FDA, Spinal muscular atrophy, MDD, Zuranolone, Failure, Biotechnology, Pathology, File, News, Risk, Drug development, Therapy, Patient, Social media, Science, Disorder, NDA, Research, Safety, Security (finance), Intellectual property, Multiple sclerosis, COVID-19, PPD, Neuroscience, Nobel Prize, Climate, Health, BIIB, Postpartum depression, Webcast, Pharmaceutical industry, Biogen, Sage

Sage Therapeutics, Inc. (Nasdaq: SAGE) and Biogen Inc. (Nasdaq: BIIB) today announced that the companies will host a joint investor webcast to discuss the planned commercialization approach and opportunity for their investigational product, zuranolone, on Tuesday, December 6 at 8:00 a.m.

Key Points: 
  • Sage Therapeutics, Inc. (Nasdaq: SAGE) and Biogen Inc. (Nasdaq: BIIB) today announced that the companies will host a joint investor webcast to discuss the planned commercialization approach and opportunity for their investigational product, zuranolone, on Tuesday, December 6 at 8:00 a.m.
  • Zuranolone is being evaluated as a potential rapid-acting, once-daily, oral two-week treatment for major depressive disorder (MDD) and postpartum depression (PPD).
  • The webcast will cover the unmet medical need in MDD, review clinical data generated with zuranolone to date and provide more detail on the potential commercialization plans and opportunity in MDD, if zuranolone is approved.
  • Sage Therapeutics is a biopharmaceutical company fearlessly leading the way to create a world with better brain health.

CANbridge Pharmaceuticals Granted Orphan Drug Designation for CAN 106 for the Treatment of Myasthenia Gravis

Retrieved on: 
Wednesday, November 16, 2022
Oncology, Health, FDA, General Health, Clinical Trials, Pharmaceutical, Biotechnology, FDA, Pompe, Weakness, Spinal muscular atrophy, Muscle contraction, Paroxysmal nocturnal hemoglobinuria, American Society of Gene and Cell Therapy, Acetylcholine, C5a, European Society for Medical Oncology, Glycogen storage disease, Ventilation, Glycogen storage disease type II, Neuromuscular disease, Degenerative disease, WuXi Biologics, University of Massachusetts, Patient, US, C3b, European Society of Gene and Cell Therapy, Muscle, Cell, C3a, SMA, Hospital, Glioblastoma, Research, MAC, Safety, Woman, Hunter syndrome, Drug, C3, Myasthenia gravis, Disease, Complement component 5, US FDA, Gene, C5, Congress, PNH, University, Haemophilia, Society, Fabry disease, Orphan, Pharmaceutical industry, Vaccine

Orphan Drug Designation for CAN106 in MG is both a validation of CANbridge innovation and a major milestone as our first US FDA regulatory designation, said James Xue, Ph.D., CANbridge Founder, Chairman and CEO.

Key Points: 
  • Orphan Drug Designation for CAN106 in MG is both a validation of CANbridge innovation and a major milestone as our first US FDA regulatory designation, said James Xue, Ph.D., CANbridge Founder, Chairman and CEO.
  • We continue to advance our global development strategy for CAN106 and look forward to developing CAN106 for myasthenia gravis and other complement-mediated diseases.
  • CAN106 is a clinical-stage investigational novel, long-acting recombinant humanized monoclonal antibody that binds to and neutralizes C5, a key component of the complement system.
  • CAN106 acts downstream of C3 in the complement pathway, preserving the generation of C3a and C3b, which are important for innate immunity.

PerkinElmer Announces its EONIS SCID-SMA Kit is First to Receive Marketing Authorization by U.S. FDA for SMA Screening in Newborns

Retrieved on: 
Monday, November 14, 2022
FDA, Other Health, Pharmaceutical, General Health, Medical Devices, Parenting, Children, Baby, Maternity, Family, Science, Biotechnology, Consumer, Other Science, Health, Research, Automation, FDA, Brainstem, Spinal muscular atrophy, Marketing, SCID, ESG, Food, Accreditation, Laboratory, Government, PerkinElmer, CE, PCR, DNA, Reproductive health, NYSE, Science, Motor neuron, SMA, Infant, Spinal cord, Software, Safety, Fundamental Analysis Software, Informatics, IVD, Birth, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, PKI, Degenerative disease, Inherited disorders of trafficking, Muscle weakness, Atrophy, Pharmaceutical industry

This is the first FDA authorized assay for SMA screening in newborns in the United States and is part of the Companys broader EONIS Platform .

Key Points: 
  • This is the first FDA authorized assay for SMA screening in newborns in the United States and is part of the Companys broader EONIS Platform .
  • This authorization is a major milestone for newborn screening in the United States.
  • Other components of the platform include the EONIS DNA Extraction kit and EONIS Analysis Software.
  • As the global leader in newborn screening, PerkinElmer offers solutions to help identify more than 50 congenital disorders.

Scholar Rock Reports Third Quarter 2022 Financial Results and Highlights Business Progress

Retrieved on: 
Monday, November 14, 2022
Biotechnology, Pharmaceutical, Health, Clinical Trials, Medicare Part D, Clinical trial, Cancer, Security (finance), Growth, Society for Immunotherapy of Cancer, Spectrochimica Acta Part B, Immunotherapy, Silicon Valley Bank, R, Medical Affairs Bureau, FAQ, Social media, SMA, Research, DRAGON, Society, CMO, RECIST, Intellectual property, Executive, COVID-19, Rock, Silicon, SAPPHIRE, Ageing, Merck, File, Annual general meeting, Spinal muscular atrophy, Drug Quality and Security Act, Human, Gongshi, Acceleron Pharma, Neurology, WMS, Fibrosis, Risk, Neuromuscular Disorders, Degenerative disease, Net, Head, SITC, Patient, Pharmaceutical industry, Palladium, Part

I am excited to strengthen our experienced executive team with the addition of Scholar Rocks new CMO, Dr. Jing Marantz.

Key Points: 
  • I am excited to strengthen our experienced executive team with the addition of Scholar Rocks new CMO, Dr. Jing Marantz.
  • Revenue was $0 for the quarter ended September 30, 2022, compared to $5.5 million for the quarter ended September 30, 2021.
  • General and administrative expense was $10.5 million for the quarter ended September 30, 2022, compared to $11.3 million for the quarter ended September 30, 2021.
  • Scholar Rock is a registered trademark of Scholar Rock, Inc.

Biogen Names Christopher Viehbacher President and Chief Executive Officer

Retrieved on: 
Thursday, November 10, 2022
Director, GLOBE, Regeneron Pharmaceuticals, Intellectual property, Health, Dupilumab, Leadership, BIIB, Partnership, Risk, Sale, Multiple sclerosis, Board, Drug discovery, Spinal muscular atrophy, Nobel Prize, Failure, NASDAQ, Share repurchase, Board of directors, U.S. Securities and Exchange Commission, Climate, Science, Neuroscience, Clinical trial, Pathology, Biotechnology, Public health, Patient, GlaxoSmithKline, Growth, Social media, Acquisition, Foundation, CNS, COVID-19, Chris Viehbacher, Management, Fine chemical, Pharmaceutical industry, Public relations, Biogen, Genzyme, Sanofi

CAMBRIDGE, Mass., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ: BIIB) today announced that its Board of Directors has appointed Christopher A. Viehbacher as President and Chief Executive Officer and a member of the Board of Directors, effective November 14.

Key Points: 
  • CAMBRIDGE, Mass., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ: BIIB) today announced that its Board of Directors has appointed Christopher A. Viehbacher as President and Chief Executive Officer and a member of the Board of Directors, effective November 14.
  • More recently, Viehbacher and Ernesto Bertarelli co-founded Gurnet Point Capital, a Cambridge-based healthcare investment fund, which has led to the creation and continuing development of many innovative companies.
  • He has also chaired or been a member of the board of a number of entrepreneurial companies in biotechnology.
  • Mr. Viehbacher said: It is both an honor and an exciting opportunity to join the Biogen team.

Survey Reveals Most People with Spinal Muscular Atrophy Using New Gene Therapies, Focused on Treatment Pipeline

Retrieved on: 
Tuesday, October 18, 2022
People, Quality of life, National Institute, National Institute of Neurological Disorders and Stroke, Muscle, SMA, Spinal cord, Drawing, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Video, Patient, Spinal muscular atrophy, Lists of diseases, Research, Motor neuron, Food, Atrium Health Union, Mutation, Industry, SMN, Weakness, CEO, Stroke, Happiness, Pharmaceutical industry, Health insurance

PHILADELPHIA, Oct. 18, 2022 /PRNewswire/ -- People with spinal muscular atrophy are relying on recently approved condition-specific treatments and are hopeful the treatment pipeline can continue to have a positive impact, according to a recent survey conducted by Health Union, the leader in social health. The inaugural Spinal Muscular Atrophy In America survey illuminates the perspectives and experiences of people living with spinal muscular atrophy.

Key Points: 
  • The inaugural Spinal Muscular Atrophy In America survey illuminates the perspectives and experiences of people living with spinal muscular atrophy.
  • Over the past six years, the U.S. Food and Drug Administration has approved the first three treatments specifically for spinal muscular atrophy.
  • In fact, three-fourths of Spinal Muscular Atrophy In America survey respondents said they currently use an "SMN-enhancing" treatment, while another 19% said they previously used one.
  • The inaugural Spinal Muscular Atrophy In America survey, which was fielded from April 19, 2021 to April 8, 2022, included responses from 54 people living with spinal muscular atrophy.

GoodRx and Biogen Collaborate to Help Enhance Enrollment Experience for Providers Who Have Chosen to Start Multiple Sclerosis Patients on VUMERITY®

Retrieved on: 
Thursday, October 13, 2022
Health, Telemedicine, Virtual Medicine, Health Technology, Health Insurance, Pharmaceutical, Biotechnology, News, U.S. Securities and Exchange Commission, Time, Risk, Health care, SEC, Nausea, MS, Science, Degenerative disease, Multiple sclerosis, Workflow, Angioedema, Infection, Nobel Prize, HCPS, NASDAQ, Liver injury, Neuroscience, Patient, Biotechnology, Diarrhea, Abdominal pain, Spinal muscular atrophy, Selected Patient Information Resources in Asian Languages, Multimedia, Technology, Private Securities Litigation Reform Act, Progressive multifocal leukoencephalopathy, Solution, Anaphylaxis, Prescription, Disability, Shingles, Acquisition, Health, Dimethyl fumarate, Climate, BIIB, HCP, Clinical trial, Excipient, Brain, Intellectual property, Hypersensitivity, Death, Failure, US Organization, COVID-19, Lists of diseases, Pathology, Flushing, View, Pharmaceutical industry, Risk management, Biogen, GoodRx

HCPs who have decided to start patients with relapsing forms of multiple sclerosis (MS) on VUMERITY (diroximel fumarate) may now find the enrollment form and submit to the specialty hub through Provider Mode .

Key Points: 
  • HCPs who have decided to start patients with relapsing forms of multiple sclerosis (MS) on VUMERITY (diroximel fumarate) may now find the enrollment form and submit to the specialty hub through Provider Mode .
  • Announced today, GoodRxs Provider Mode is a new experience built specifically for HCPs to help them access solutions for the medications they prescribe.
  • We are proud to work with medical professionals to help patients with relapsing MS get the therapy they need more efficiently.
  • This exciting integration with Biogen is just the start of the great work to come as we expand our offerings for providers.

Scholar Rock to Present New 24-Month Data from Phase 2 TOPAZ Study at Two International Conferences: World Muscle Society Congress and International Scientific Congress on Spinal Muscular Atrophy

Retrieved on: 
Tuesday, October 4, 2022
Biotechnology, General Health, Health, Pharmaceutical, Clinical Trials, Cancer, Review, Gene, Clinical trial, Private Securities Litigation Reform Act, Drug Quality and Security Act, SMN1, Growth, SMN2, U.S. Securities and Exchange Commission, Gongshi, Security (finance), Genetic disorder, Social media, EMA, Therapy, Atrophy, SMN, NASDAQ, Neuromuscular Disorders, Myostatin, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Investor, Pathology, Time, SMA, Poster, Patient, European Medicines Agency, Human, Muscle weakness, Survival, Food, Johns Hopkins University, Twitter, Fibrosis, FDA, File, Publication, Interim, FAQ, Rock, Motor neuron, Company, LinkedIn, Risk, TOPAZ, Spinal muscular atrophy, Neurology, WMS, Safety, Pediatrics, Fast Track, Securities Act of 1933, Pharmaceutical industry, Medical imaging

Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA).

Key Points: 
  • Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA).
  • Scholar Rock believes that inhibiting myostatin activation with apitegromab may result in a clinically meaningful improvement in motor function in patients with SMA.
  • Spinal muscular atrophy (SMA) is a rare, and often fatal, genetic disorder that typically manifests in young children.
  • It is characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk and progressive muscle weakness.

Biogen and Denali Therapeutics Announce Initiation of the Phase 3 LIGHTHOUSE Study in Parkinson’s Disease Associated with LRRK2 Pathogenic Mutations

Retrieved on: 
Monday, October 3, 2022
Nasdaq, Private Securities Litigation Reform Act, Form 10-K, Failure, COVID-19, Traffic barrier, U.S. Securities and Exchange Commission, Therapy, Security (finance), Intellectual property, Climate, Research, Health, Nobel Prize, Drug development, Veni, vidi, vici, Mutation, Pathology, Science, Biotechnology, Patient, Biomarker, Multiple sclerosis, Genotype, BIIB, Lists of diseases, SEC, Neurodegeneration, Spinal muscular atrophy, The Lancet, Forward-looking statement, Risk, GLOBE, Neuroscience, Biology, Toxic shock syndrome, Disease, BBB, Phenotype, LUMA, Safety, Unified, ATV, Pharmaceutical industry, Vaccine, LRRK2, Biogen

The LIGHTHOUSE study will specifically recruit individuals with a pathogenic mutation in LRRK2, enabling us to test the genetic hypothesis and implicated lysosomal pathway.

Key Points: 
  • The LIGHTHOUSE study will specifically recruit individuals with a pathogenic mutation in LRRK2, enabling us to test the genetic hypothesis and implicated lysosomal pathway.
  • The LIGHTHOUSE study is the largest study ever undertaken in individuals with Parkinsons disease caused by a LRKK2 mutation.
  • Denali and Biogen are co-developing and co-commercializing BIIB122 for the potential treatment of Parkinsons disease.
  • The initiation of the Phase 3 LIGHTHOUSE study marks an important milestone in the BIIB122 development program.