Muscular Health News
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Advances in Newborn SMA Screening research by SNP Biotechnology
BSD Robotics is proud to share a recent study conducted by SNP Biotechnology R&D Ltd “A new line method; A direct test in spinal muscular atrophy screening for DBS” now published in the journal of Molecular Genetics and Genomic Medicine. Given spinal muscular atrophy (SMA) is one of the most prevalent genetic disorders affecting young children and a major cause of death in ...
By BSD Robotics
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CD Genomics Unveils Advanced Mitochondrial Diseases Panel Sequencing Service
CD Genomics, a leading biotechnology company specializing in genomics and sequencing, has announced the launch of its advanced Mitochondrial Diseases Panel Sequencing services. This new panel enables rapid and accurate detection of mutations in mitochondrial DNA (mtDNA) that are associated with a wide range of mitochondrial disorders. Mitochondrial diseases are a complex and heterogeneous group ...
By CD Genomics
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Roswell Biotechnologies Forms Scientific Advisory Board
Roswell Biotechnologies, Inc., the molecular electronics company, announced today the formation of its Scientific Advisory Board (SAB). Comprised of industry and academic leaders in science and technology, the new SAB will be instrumental in the final stages of development and commercialization of the company's molecular electronics chip, a fully scalable universal biosensor capable of seeing ...
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Sage Therapeutics and Biogen Present Further Analyses from Phase 3 SKYLARK Study of Zuranolone in Postpartum Depression at the European College of Neuropsychopharmacology (ECNP) Congress
Zuranolone 50 mg demonstrated a clinically meaningful and statistically significant improvement in depressive symptoms at Day 15, the primary endpoint, and at Days 3, 28, and 45, key secondary endpoints as previously reported Newly presented data offered additional insight into the SKYLARK Study and further demonstrated the rapid improvements in depressive symptoms observed in the clinical ...
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Sage Therapeutics and Biogen to Host Investor Webcast on December 6, 2022 to Discuss Potential Commercialization Plans for Zuranolone
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. ET. Zuranolone is being evaluated as a potential rapid-acting, once-daily, oral two-week treatment for major ...
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MDA Venture Philanthropy Grant to Support Development of ALS Biomarkers
The Muscular Dystrophy Association is pleased to announce the award of an MDA Venture Philanthropy (MVP) grant totaling $233,200 to Scottsdale, Ariz.-based Iron Horse Diagnostics to support development of a prognostic test for ALS (amyotrophic lateral sclerosis). Such a test could help improve and accelerate clinical trials and speed the development of life-saving drugs to people with ALS. MVP ...
By nVector
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Forge Biologics Announces Updated Positive Clinical Data in RESKUE, a Novel Phase 1/2 Gene Therapy Trial for Patients with Krabbe Disease
Clinical data demonstrating initial safety and efficacy from the RESKUE trial are being presented by Chief Medical Officer Maria Escolar, M.D., at the 29th Congress of European Society of Gene & Cell Therapy (ESGCT), October 11-14, 2022 Subjects treated with FBX-101 have shown increased galactocerebrosidase (GALC) enzyme activity in plasma and cerebrospinal fluid (CSF), normal white matter ...
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Sage Therapeutics and Biogen Present New Analyses at Psych Congress Further Evaluating the Efficacy and Safety of Zuranolone
Sage Therapeutics, Inc. (Nasdaq: SAGE) and Biogen Inc. (Nasdaq: BIIB) today announced new analyses from across the development program for zuranolone, an investigational, oral, once-daily, 14-day treatment in clinical development for adult patients with major depressive disorder (MDD) and postpartum depression. The 11 new analyses are being presented at the 2022 Psych Congress in New Orleans, ...
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Forge Reports Positive Clinical Data on Brain Development and Motor Function from the RESKUE Novel Phase 1/2 Gene Therapy Trial in Patients with Krabbe Disease at the SSIEM Annual Symposium
FBX-101 clinical data demonstrating safety and initial efficacy from the RESKUE trial is being presented at the 2022 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium FBX-101 is a systemic AAV gene therapy following unrelated cord blood transplantation (“UCBT”) and has been well tolerated through Day 180 FBX-101 significantly increased GALC enzyme ...
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August 2022 is Spinal Muscular Atrophy Awareness Month!
Every year, the month of August is nationally recognized as Spinal Muscular Atrophy (SMA) Awareness Month. This month-long awareness campaign serves as an important time to demonstrate support and help promote awareness for this rare disease affecting lives across the world. WHAT IS SMA? Affecting 4 people in every 100,000 in the U.S., Spinal Muscular Atrophy (SMA) is a genetic, progressive ...
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FireGene's Monkeypox Virus Nucleic Acid Detection Kit (Real-time PCR Method) Receives CE-IVDD Certification
FireGene announces that its Monkeypox Virus Nucleic Acid Detection Kit (Real-time Fluorescent PCR Method) has recently obtained CE-IVDD approval. This RT-PCT kit can be used to detect suspected cases of MPV infection. Rash fluid, nasopharyngeal swabs, pharyngeal swabs, and serum samples are applicable for the test. The test result is available in 40 minutes after sample collection. This kit is ...
By FireGene
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New Startup Makes Developing Gene Therapies Faster and Easier
Today, cell and gene therapies treat and could even cure terminal diseases like leukemia and spinal muscular atrophy, but unlocking their full potential is still a challenge for therapy developers. Both types of therapies rely on living cells comprised of hundreds of thousands of biomolecules to work in unison. And in order for therapy developers to meet safety and production ...
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Vertex to Present New Data on VX-880 Clinical Trial at 82nd American Diabetes Association Annual Conference
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced three abstracts, including two oral presentations and one poster, detailing data on type 1 diabetes (T1D) and its Phase 1/2 trial of VX-880, a stem cell-derived, fully differentiated pancreatic islet cell replacement therapy, have been accepted for presentation during the American Diabetes Association 82nd Annual Scientific ...
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TikoMed AB reports positive phase II clinical data for the treatment of Amyothropic Lateral Sclerosis in data published by PLOS ONE
A new study with TikoMed’s platform lead clinical drug candidate ILB®, provides positive safety and tolerability results and the first clinical evidence of encouraging clinical efficacy of ILB® in the treatment of Amyotrophic Lateral Sclerosis (ALS). The study was published in PLOS ONE today. In a phase II study of ILB administered to patients with ALS for five weeks, the ...
By TIKOMED AB
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Capricor Therapeutics Appoints Xavier Avat as Chief Business Officer
Capricor Expands Senior Management Team to Build Commercial Strategy for Lead Asset, CAP-1002, and Further Develop Partnering Strategy for Proprietary Exosome Platform Formerly at Edwards Lifesciences, Gilead and Genentech, Mr. Avat Brings Over 25 Years of Business and Commercial Strategy Experience to Capricor Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company focused on the ...
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Capricor Therapeutics to Present at the H.C. Wainwright Global Investment Conference
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, announced today that Dr. Linda Marbán, CEO of Capricor, will present at the H.C. Wainwright Global Investment Conference being held on May 23-26, 2022 in Miami, Florida. Please see ...
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Suketu Upadhyay Elected to Vertex Board of Directors
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Suketu (“Suky”) Upadhyay has been elected to its board of directors as an independent director. Mr. Upadhyay is a global business executive with more than 20 years of experience in the pharmaceutical and medical technology sectors, serving in financial and strategy roles covering multiple areas of the life ...
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AVROBIO Announces Preclinical Gene Therapy Data for Pompe Disease at American Society of Gene and Cell Therapy (ASGCT) Annual Meeting
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced preclinical data demonstrating the efficacy and safety of AVR-RD-03 gene therapy in a mouse model of infantile onset Pompe disease. The results, which will be presented today during the “Disease models and Clinical Applications: ...
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Vertex Announces Further Expansion in the Boston Seaport at Dedication of the Jeffrey Leiden Center for Cell and Genetic Therapies
New 344,000 square foot cell and genetic therapies research and manufacturing facility to be built Facility to support continued R&D growth At a dedication today of the new Jeffrey Leiden Center for Cell and Genetic Therapies in Boston’s Seaport, Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced plans to build another 344,000 square foot facility in the Seaport to ...
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Vertex Announces $50 Million Commitment to Health Equity With Three Initial Grants
Vertex grant to JDRF supports efforts to improve clinical trial diversity in type 1 diabetes The Vertex Foundation grant supports the Mass General Comprehensive Sickle Cell Disease Treatment Center Additional Vertex Foundation grant to Year Up to support health care workforce training Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Vertex and the Vertex Foundation, ...
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