Spinal muscular atrophy

SOLVE FSHD invests US$3 million in Altay Therapeutics

Retrieved on: 
Wednesday, September 21, 2022
SOLVE, SMA, Health, Human, DUX4, Ageing, Biomedical Research, Entrepreneurship, Economics, Chip, Life, Harvard Business School, Myasthenia gravis, Therapy, Amyotrophy, Board, Patient, Facioscapulohumeral muscular dystrophy, University of Oslo, CEO, Technology, MBA, Leadership, Drug development, Spinal muscular atrophy, Y Combinator, ALS, Research, GLOBE, Amyotrophic lateral sclerosis, University, Cancer, Disease, Pharmaceutical industry, Medical device, Management, BC, FSHD

We are excited to partner with Altay Therapeutics and help accelerate the discovery and development of much-needed therapies for FSHD.

Key Points: 
  • We are excited to partner with Altay Therapeutics and help accelerate the discovery and development of much-needed therapies for FSHD.
  • Altay Therapeutics unique platform technology will help to identify and accelerate novel and targeted treatments for FSHD, says Dr. Eva Chin, Executive Director, SOLVE FSHD.
  • SOLVE FSHDs targeted investment into Altay Therapeutics will support the companys growth and will help identify a DUX4 inhibitor for clinical trials.
  • Altay Therapeutics Board of Directors will be led by Mr. Eric Bjerkholt , Chairman, with Dr. Eva Chin , Executive Director for SOLVE FSHD and Dr. Ali Ozes , Co-Founder and CEO of Altay Therapeutics, as directors.

Scholar Rock Appoints Jay Backstrom, M.D., M.P.H., to be its Next Chief Executive Officer

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Tuesday, September 20, 2022
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Safety, Risk, SMA, FAQ, Human, COVID-19, Nagesh, Twitter, Fibrosis, Research, Securities Act of 1933, Â, Hoechst AG, Temple University Hospital, LinkedIn, Company, Part, Review, Private Securities Litigation Reform Act, Spinal muscular atrophy, Social media, Growth, Investor, Temple University School of Medicine, U.S. Securities and Exchange Commission, Patient, File, Clinical trial, Cancer, CEO, IQVIA, Interim, Acceleron Pharma, Internal medicine, Board of directors, Leadership, Intellectual property, NASDAQ, Rock, Board, Security (finance), R, Multimedia, Celgene, Merck, Neuromuscular Disorders, Public health, Management, Pharmaceutical industry, Director, Gongshi

He most recently served as Executive Vice President, Research and Development at Acceleron Pharma, which was acquired by Merck in 2021.

Key Points: 
  • He most recently served as Executive Vice President, Research and Development at Acceleron Pharma, which was acquired by Merck in 2021.
  • Jay is an exceptional executive leader with a track record of success that spans several decades in the biopharmaceutical industry.
  • I would like to thank Nagesh for his remarkable leadership and commitment to Scholar Rock during his 10-year tenure at the company.
  • Scholar Rock is a registered trademark of Scholar Rock, Inc. REBLOZYL is a registered trademark of Celgene Corporation.

Registration Now Open for 2023 MDA Clinical & Scientific Conference, to Showcase Cutting Edge Research Advancements and Clinical Achievements in Neuromuscular Disease

Retrieved on: 
Monday, September 12, 2022
YouTube, Limb–girdle muscular dystrophy, SMA, Myotonic dystrophy, Glycogen storage disease type II, NMAC, Congenital muscular dystrophy, COVID-19, Muscular Dystrophy Association, Twitter, MDA, FDA, CRISPR, MG, Knowledge, NEW, Organization, LinkedIn, Hilton Anatole, Public policy, Spinal muscular atrophy, Neuromuscular disease, Myasthenia gravis, History, Disease, CEO, Technology, 2021 Essex County Council election, CMT, CMD, LGMD, Doctor of Philosophy, Charcot–Marie–Tooth disease, Episcopal conference, Amyotrophic lateral sclerosis, Degenerative disease, Muscular dystrophy, TikTok, ALS, Research, Lists of diseases, Conference, DMD, Medicine, Multimedia, DM, Duchenne muscular dystrophy, Pharmaceutical industry, Dentistry, Public relations, Facebook, Instagram

NEW YORK, Sept. 12, 2022 /PRNewswire/ -- The Muscular Dystrophy Association (MDA) today announced that registration is open for the 2023 MDA Clinical & Scientific Conference being held March 19-22, 2023. MDA is the largest worldwide convener of the neuromuscular disease community of renowned researchers, clinicians, academicians, advocates, and industry leaders both in person at the Hilton Anatole in Dallas, Texas and via live stream.

Key Points: 
  • What is equally important is that we convene MDA's Care Center Network of neuromuscular disease specialists, connecting them to the latest clinical trials and FDA approved treatments.
  • They are also developing new treatments and creating the pipeline of progress that is the hallmark of today's neuromuscular disease frontier."
  • Before the conference, MDA will once again host the annual gathering of the Neuromuscular Advocacy Collaborative (NMAC).
  • For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families.

Voyager Therapeutics Appoints Peter Pfreundschuh as CFO; Todd Carter, Ph.D., as Chief Scientific Officer; and Trista Morrison as SVP Corporate Affairs

Retrieved on: 
Wednesday, September 7, 2022
AstraZeneca, Technology, Science, U.S. Securities and Exchange Commission, Harvard University, GLOBE, Leadership, Biology, Company, Broad Institute, Lists of diseases, Patient, B.A, Salk Institute for Biological Studies, Corporate Affairs, Genetics, Government, Sale, SVP, MIT, Private Securities Litigation Reform Act, Senior, Texas A&M University, CSO, CFO, Nasdaq, FDA, Rutgers University, Rider University, Johnson & Johnson, Industry, Morrison, AAV, Frequency, PR, Security (finance), Corporation, Therapy, Spinal muscular atrophy, LLP, R, Tulane University, Public relations, MBA, Drug development, Marketing, Ernst & Young, Columbia University, Biomedical Research, Communication, Publication, Annual report, CNS, Management, Pharmaceutical industry, Voyager, Sucampo Pharmaceuticals, Research, Intention, Mallinckrodt

Petes extensive corporate finance experience will be integral in maximizing the value of our AAV capsid platform and our pipeline.

Key Points: 
  • Petes extensive corporate finance experience will be integral in maximizing the value of our AAV capsid platform and our pipeline.
  • Peter Pfreundschuh hasmore than 25 years of finance, business development, commercial and public company leadership experience in the life sciences and medical device industries.
  • Trista Morrison has more than 20 years of experience in healthcare public affairs, including strategic communications, investor relations and patient advocacy.
  • Pfreundschuh, Carter and Morrison will report to Voyager CEO Al Sandrock, M.D., Ph.D., and will serve on Voyagers Senior Leadership Team.

Alpha Cognition Announces Second Quarter 2022 Results and Provides Corporate Update

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Thursday, August 25, 2022
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Alpha Cognition Inc. (TSX-V: ACOG) (OTCQB: ACOGF) (Alpha Cognition, or the Company), a biopharmaceutical company committed to developing novel therapies with the potential to transform the lives of people with debilitating neurodegenerative disorders, today reported financial results and provided a corporate update for the second quarter ended June 30, 2022.

Key Points: 
  • Alpha Cognition Inc. (TSX-V: ACOG) (OTCQB: ACOGF) (Alpha Cognition, or the Company), a biopharmaceutical company committed to developing novel therapies with the potential to transform the lives of people with debilitating neurodegenerative disorders, today reported financial results and provided a corporate update for the second quarter ended June 30, 2022.
  • "The Company released positive topline data from the ALPHA-1062 pivotal trial during the second quarter of 2022 which allows for the preparation of an NDA filing for the product.
  • The Company is seeking additional capital to fund this study initiation and will commence the study within a quarter to securing the required funding.
  • R&D expenses increased in 2022 primarily due to the additional costs associated with advancing ALPHA-1062 and ALPHA-0602 clinical and preclinical studies.

Sanofi appoints new Heads of U.S. General Medicines, and North America Vaccines and Consumer Healthcare

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Wednesday, August 3, 2022
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BRIDGEWATER, N.J., Aug.3, 2022 /PRNewswire/ -- Sanofi announces three appointments to key leadership roles for U.S. General Medicines and its North American Vaccines and Consumer Healthcare businesses.

Key Points: 
  • BRIDGEWATER, N.J., Aug.3, 2022 /PRNewswire/ -- Sanofi announces three appointments to key leadership roles for U.S. General Medicines and its North American Vaccines and Consumer Healthcare businesses.
  • Deborah Glasser is the new Head of Vaccines, North America, responsible forthe company's industry-leading vaccines business in the U.S. and Canada.
  • Andrew Loucks will take over as Head of North America Consumer Healthcare, effective August 8.
  • Loucks joins Sanofi from Sensory Cloud Inc., where he was CEO and a Board member of the Boston-based consumer health & technology company.

IAMA Therapeutics Announces Initiation of Preclinical Development Studies in Drug-resistant Epilepsy Under a Service Agreement with Psychogenics

Retrieved on: 
Monday, July 25, 2022
Health, Consumer, Neurology, Children, Clinical Trials, Pharmaceutical, Biotechnology, SMA, Mark, EEG, Seizure, Translational neuroscience, Parkinson's disease, Research, Muscular dystrophy, CRO, Drug discovery, Lists of diseases, Alzheimer's disease, Partnership, Therapy, Anticonvulsant, Autism spectrum, Spinal muscular atrophy, Autism, Biotechnology, Epilepsy, MBA, Huntington's disease, GABAergic, Sleep, Electrophysiology, Molecular biology, IAMA, Neuroscience, Psychology, Pharmaceutical industry

"PsychoGenics is a leader in preclinical behavioral neurobiology with a successful track record of anti-epilepsy drug testing through translational EEG approaches, including electrographic and behavioral identification of seizure," said Andrea P. Malizia, Ph.D., MBA, Chief Executive Officer of IAMA Therapeutics.

Key Points: 
  • "PsychoGenics is a leader in preclinical behavioral neurobiology with a successful track record of anti-epilepsy drug testing through translational EEG approaches, including electrographic and behavioral identification of seizure," said Andrea P. Malizia, Ph.D., MBA, Chief Executive Officer of IAMA Therapeutics.
  • We are excited to embark of these studies with IAMA Therapeutics said Stephen Morairty, Ph.D., Vice President of Translational Neuroscience at PsychoGenics.
  • Using our pre-clinical MTLE model, will carefully evaluate the efficacy of these first-in-class selective-NKCC1 inhibitors.
  • IAMA Therapeutics is a preclinical-stage pharmaceutical company focused on discovering, developing, and commercializing novel medicines to make a difference in the lives of children and adults suffering from epilepsy, neurodevelopmental and cognitive disorders.

McKinsey expands network of Digital Capability Centers in partnership with New Jersey Innovation Institute (NJII) with advanced biopharma manufacturing learning facility in Newark

Retrieved on: 
Saturday, July 16, 2022
Learning, CEO, Patient, Method, Solution, Artificial intelligence, Center, Adoption, Spinal muscular atrophy, McKinsey & Company, Leber congenital amaurosis, CGT, Regenerative medicine, Organization, Blog, Medicine, Garden, Company, Growth, NJ, Partnership, New Jersey Institute of Technology, Garden State, Workforce, Television, Technology

NEWARK, N.J., July 15, 2022 /PRNewswire/ -- McKinsey & Company launched a new high-tech learning and innovation facility today in the Garden State to support the advancement of cell and gene therapy (CGT) manufacturing, expanding its global network of Digital Capability Centers.

Key Points: 
  • In partnership with the New Jersey Innovation Institute (NJII) and operated by BioCentriq, McKinsey's Newark-based facility specializes in innovative manufacturing processes in life sciences.
  • This facility will help biopharma companies sustain higher levels of innovation and accessibility by helping to solve their operational challenges."
  • In addition to the 250 McKinsey experts involved in this global network, the Digital Capability Center network also has partnerships with 150+ tech companies and startups.
  • The Digital Capability Center New Jersey will be located on the Newark campus of the New Jersey Institute of Technology.

Vibrent Health Digital Health Research Platform Optimizes Accessibility and User Experience for People with Disabilities to Increase Diverse Participation in Research

Retrieved on: 
Wednesday, June 29, 2022
Pharmaceutical, Physical Therapy, General Health, Health, Mental Health, Data Management, Health Technology, Technology, Rehabilitation, Movement, CEO, Spinal muscular atrophy, Health Level Seven International, World Wide Web Consortium, Research, Precision medicine, Biomedical Research, Engineering, Health, ICT, Population, App Store (iOS/iPadOS), WAI, AA, National, ADA, WCAG, Compliance, NIH, Rehabilitation Act of 1973, Technology, Section 508 Amendment to the Rehabilitation Act of 1973, PAB, Disability discrimination act, Congress, Disability, Information technology, Program, Data collection, Medical device, Online shopping

Vibrent Health , a health technology startup, is helping researchers recruit and engage people with disabilities in research by ensuring that digital health research tools the company develops support people with disabilities and comply with government rules for accessibility.

Key Points: 
  • Vibrent Health , a health technology startup, is helping researchers recruit and engage people with disabilities in research by ensuring that digital health research tools the company develops support people with disabilities and comply with government rules for accessibility.
  • Making digital health technology products that accommodate people with disabilities can help researchers reach their inclusion goals and obtain more complete data by including diverse participants.
  • Recruiting diverse individuals has become increasingly important in health research and can be a prerequisite for research sponsorship and funding.
  • Vibrent Health develops digital health technology and research tools for health organizations, researchers and research participants.

Biogen and Happify Health Collaborate to Support Multiple Sclerosis Patients on Digital Platform

Retrieved on: 
Wednesday, June 22, 2022
Data Management, Health, Technology, Other Health, Software, Internet, Well, Multiple sclerosis, Â, Digital, Pathology, Community, Education, Intellectual property, Lists of diseases, Therapy, COVID-19, Practitioner, Spinal muscular atrophy, Disease management, Science, Nobel Prize, Intelligence, Neuroscience, Artificial intelligence, Woman, Nursing, Digital therapeutics, Empathy, U.S. Securities and Exchange Commission, Biotechnology, Leadership, Private Securities Litigation Reform Act, Attack, Neurodegeneration, Disease, Physician, National, Failure, NASDAQ, Health, Climate, MS, News, Partnership, Patient, Multimedia, Alternative, Psoriasis, Risk, BIIB, Medical device, Drug, Pharmaceutical industry, Risk management, Biogen

Biogen (NASDAQ:BIIB) and Happify Health ( www.HappifyHealth.com ), the Intelligent Healing Company, today announced a collaboration to provide a digital solution for patient education and engagement, powered by artificial intelligence (AI), to support people living with multiple sclerosis (MS).

Key Points: 
  • Biogen (NASDAQ:BIIB) and Happify Health ( www.HappifyHealth.com ), the Intelligent Healing Company, today announced a collaboration to provide a digital solution for patient education and engagement, powered by artificial intelligence (AI), to support people living with multiple sclerosis (MS).
  • This collaboration is one of many ways we are focused on supporting patients by meeting them where they are.
  • We are excited about the potential of this partnership to provide overall support for the MS community.
  • Happify Health uses its proprietary AI technology to personalize content to patients life stages, symptoms and interests.