Spinal muscular atrophy

Genentech Announces Industry-Leading Brain Health Research Collaborations and Latest Data Across Neuroscience Medicines at AAN 2023 Annual Meeting

Retrieved on: 
Monday, April 24, 2023
Mental Health, Health, Telemedicine, Virtual Medicine, Neurology, Clinical Trials, Pharmaceutical, Biotechnology, DSM-IV codes, Spinal muscular atrophy, Survival, Multiple sclerosis, Science, Patient, Nervous system, Ageing, Policy, IHME, Disease, Maintenance, Cleveland Clinic, University, Disability, Cardiovascular disease, Dementia, Brain, NMOSD, Research, Satralizumab, RO, RMS, RRMS, Cleveland, Immunodeficiency, Phase, Death, Safety, Huntington's disease, Abstract, Natural history, COVID-19, Multimedia, ROG, Brainpower, Neuroscience, Myasthenia gravis, Cancer, EMPOWER, Angelman syndrome, SMA, Experience, Telehealth, Partnership, Central nervous system, AAN, American Academy, Digital technology, Annual general meeting, Neuromyelitis optica spectrum disorder, Tenecteplase, AD, Pharmaceutical industry, Medical imaging, Risdiplam, Genentech

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), is announcing three new partnerships focused on improving brain health outcomes.

Key Points: 
  • Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), is announcing three new partnerships focused on improving brain health outcomes.
  • “With that in mind, we are committed to developing and delivering new medicines that address areas of highest need in neuroscience.
  • See AAN oral presentation details on Genentech’s telehealth pilot with Cleveland Clinic here , Tuesday, April 25 at 3:54 p.m.
  • To meet the needs of people living with brain health conditions, Genentech is continuing to grow and advance its pipeline, with key areas of focus in neuroimmunologic, neuromuscular, neurodegenerative and neurodevelopmental diseases.

Biophytis Announces 2022 Financial Results and 2023 Perspectives

Retrieved on: 
Tuesday, April 18, 2023
EUA, Amount of substance, EAP, Euronext Growth, Duchenne muscular dystrophy, SARA, CMA, Therapy, CET, Marketing, Bank statement, AMD, COVID-19, Clinical trial, Ageing, Research, MACA, Trial of the century, EMA, FAMHP, Death, Spinal muscular atrophy, Investigational New Drug, DMD, Patient, Risk, AMF, 237th Rifle Division, FDA, Phase, IND, Respiratory failure, Safety, Cryptocurrency, Pharmaceutical industry, Dietary supplement, SMA, Sarcopenia

€11.1M of available cash and equivalents on December 31, 2022, and financing instruments for a total of €22M allowing financial visibility beyond mid 2024

Key Points: 
  • €11.1M of available cash and equivalents on December 31, 2022, and financing instruments for a total of €22M allowing financial visibility beyond mid 2024
    Paris, France, Cambridge (Massachusetts, United States), April 18th, 2022 – 8am CET - Biophytis SA (NasdaqCM: BPTS, Euronext Growth Paris: ALBPS), (“Biophytis” or the “Company”), a clinical-stage biotechnology company focused on the development of therapeutics that slow the degenerative processes associated with aging, including severe respiratory failure in patients suffering from COVID-19, today announces its financial results for the year ended December 31, 2022, and provides updates on key perspectives for 2023.
  • Stanislas Veillet, President and CEO of Biophytis, stated: “In a difficult economic and financial environment, 2022 was a crucial year for Biophytis.
  • Scientific communication in international congresses of the main results of the SARA (Sarcopenia) and MYODA (Duchenne Muscular Dystrophy) programs.
  • The Company’s annual 2022 non-audited consolidated financial statements prepared in accordance with IFRS were reviewed by the Company’s Board of Directors on April 14, 2023.

CENTOGENE Launches NEW CentoGenome®, World’s Most Comprehensive Whole Genome Sequencing Solution for Diagnosis of Rare and Neurodegenerative Diseases

Retrieved on: 
Wednesday, April 12, 2023
SMA, PD, Genomics, Health care, NGS, DNA sequencing, Gaucher's disease, GD, DSM-IV codes, CNV, Whole genome sequencing, Spinal muscular atrophy, Patient, Physician, Combine, WGS, Diagnosis, Polymerase chain reaction, Bias, PCR, Genome, Medical device, Medical imaging

and ROSTOCK, Germany and BERLIN, April 12, 2023 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced the launch of NEW CentoGenome®, an enhanced Next Generation Sequencing (NGS)-based assay.

Key Points: 
  • and ROSTOCK, Germany and BERLIN, April 12, 2023 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced the launch of NEW CentoGenome®, an enhanced Next Generation Sequencing (NGS)-based assay.
  • Healthcare professionals can leverage NEW CentoGenome to provide more comprehensive diagnostic information, which could accelerate access to potential treatment options.
  • Serving as a first-line test, NEW CentoGenome is the most comprehensive commercially available Whole Genome Sequencing (WGS) test on the market for both rare and neurodegenerative disorders – covering almost all disease-causing variants, including the most relevant repeat expansions associated with neurological diseases, in a single assay.
  • “Building on this expertise, CENTOGENE’s enhanced whole genome sequencing is the leading solution on the market, reflecting the latest advanced technologies and unique insights that can’t be found anywhere else to provide maximized disease coverage.

VintaBio Unveils $64M to Back Viral Vector Trailblazers Behind the First Approved Cell and Gene Therapies

Retrieved on: 
Tuesday, April 11, 2023
Analytical chemistry, ILC Dover, University, Spinal muscular atrophy, Eli Lilly and Company, Thermo Fisher Scientific, Child, Doctor of Philosophy, Eli Lilly, CDMO, Patient, Voretigene neparvovec, Hospital, FDA, Children's Hospital of Philadelphia, Vaccine, Liu Junwei, Sanofi

VintaBio was founded by Junwei Sun and Dr. Shangzhen Zhou, who developed the first viral vectors used for life-saving therapies, including the cell therapy that cured Emily Whitehead of a pediatric cancer, as well as the first two FDA-approved gene therapies.

Key Points: 
  • VintaBio was founded by Junwei Sun and Dr. Shangzhen Zhou, who developed the first viral vectors used for life-saving therapies, including the cell therapy that cured Emily Whitehead of a pediatric cancer, as well as the first two FDA-approved gene therapies.
  • "I co-founded VintaBio to make sure viral vectors never prevent someone from receiving a life-changing treatment.
  • "With VintaBio, we now want to provide the ability to scale cutting-edge therapies for thousands more in-need patients."
  • Radspinner joins VintaBio from ILC Dover, a New Mountain Capital portfolio company, where he served as president for the organization's biotherapeutics portfolio.

BioDuro-Sundia Announces Formation of Scientific Advisory Board

Retrieved on: 
Monday, March 27, 2023
Biotechnology, Health, Science, Pharmaceutical, Research, Molecule, SAB, Medicinal chemistry, University, ARCH Venture Partners, Denali, Berkeley, Stanford University, Harvard University, London Business School, Health care, Roche, IMD, Interline, Book, Spinal muscular atrophy, Novartis, Drug discovery, Advent International, Growth, Therapy, NIH, Pharmaceutical industry, Genentech, Chemistry

BioDuro-Sundia, a leading drug discovery, development and commercial services organization backed by Advent International, announced today the establishment of a Discovery Research Scientific Advisory Board (SAB) with the appointment of Andrew Thomas Ph.D. and Zach Sweeney Ph.D. as its members.

Key Points: 
  • BioDuro-Sundia, a leading drug discovery, development and commercial services organization backed by Advent International, announced today the establishment of a Discovery Research Scientific Advisory Board (SAB) with the appointment of Andrew Thomas Ph.D. and Zach Sweeney Ph.D. as its members.
  • “We welcome Andrew and Zach who bring valuable experience as we continue to accelerate our growth,” said Dr. Kent Payne, CEO of BioDuro-Sundia.
  • Zach was previously the Chief Scientific Officer at Denali Therapeutics and a leader in the drug discovery groups at Revolution Medicines, Novartis, Genentech, and Roche.
  • He is listed as an inventor on approximately 60 published patent applications and has co-authored more than 40 scientific publications.

Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset

Retrieved on: 
Sunday, March 19, 2023
Failure mode and effects analysis, Non-invasive ventilation, SMA, Interim, Gastroenteritis, SMN2, IV, Spinal muscular atrophy, Observational study, STRONG, Marketing, Dehydration, Survival, Clinical trial, Quality of life, Commonwealth of Australia (US securities entity), Research, Ventilation, Government, Sale, Walking, MD, Patient, Muscular Dystrophy Association, Regulation, Physician, Pandemic, Episcopal conference, Common, Viral, Diagnosis, MDA, Periodic breathing, Nationwide, Conference, Pneumonia, Safety, Disease, Birth control, Pharmaceutical industry, Nursing, Medical device, Sales, COVID-19, Novartis, Onasemnogene abeparvovec

“These children now have an improved quality of life, vastly different from what would have been expected for them if they had not received treatment.

Key Points: 
  • “These children now have an improved quality of life, vastly different from what would have been expected for them if they had not received treatment.
  • Results from the IV cohort, which included 63 patients, demonstrated how a single administration of Zolgensma provided consistent, substantial and durable efficacy over time.
  • “Data from the LT-001 and LT-002 studies showed that, regardless of the patient’s symptomatic status at the time of treatment, Zolgensma IV is an effective and durable treatment option.
  • Nor can there be any guarantee that such products will be commercially successful in the future.

Caroline Dorsa to Succeed Stelios Papadopoulos as Chair of Biogen Board of Directors

Retrieved on: 
Wednesday, March 8, 2023
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(NASDAQ: BIIB) today announced that the Board of Directors has elected Caroline Dorsa as Chair of the Board of Directors, effective immediately following the Company’s 2023 Annual Meeting of Stockholders (the “Annual Meeting”), scheduled to take place on June 14, 2023.

Key Points: 
  • (NASDAQ: BIIB) today announced that the Board of Directors has elected Caroline Dorsa as Chair of the Board of Directors, effective immediately following the Company’s 2023 Annual Meeting of Stockholders (the “Annual Meeting”), scheduled to take place on June 14, 2023.
  • Ms. Dorsa will succeed Stelios Papadopoulos, Ph.D., who announced he would not stand for reelection to the Board at the Annual Meeting.
  • Dr. Papadopoulos said: “The entire Board has great respect for Caroline and the tremendous contributions she has made during her 13-year tenure and as Chair of our Audit Committee, where she has exhibited exceptional judgement, integrity, and dedication.
  • I’m stepping down knowing that the Board will be in very good hands under Caroline’s outstanding leadership.”
    Ms. Dorsa joined the Biogen Board in 2010.

Plasmid DNA Contract Manufacturing Global Market Report 2023: Sector to Reach $1.1 Billion by 2030 at a CAGR of 19.3% - ResearchAndMarkets.com

Retrieved on: 
Tuesday, March 7, 2023
Biotechnology, Genetics, Health, Vaccine, Incidence, Prevalence, Research, Viral, Growth, Chapter Three, Drugs Controller General of India, DNA, Malignancy, Entrepreneurship, Spinal muscular atrophy, Cancer, Immunotherapy, COVID-19, Safety, Conditional sentence

Hence, the growing adoption of gene therapy in the treatment of cancer is propelling the growth of the plasmid DNA contract manufacturing market.

Key Points: 
  • Hence, the growing adoption of gene therapy in the treatment of cancer is propelling the growth of the plasmid DNA contract manufacturing market.
  • However, the COVID-19 resulted into positive impact on the plasmid DNA contract manufacturing market.
  • The market would certainly benefit from the efforts of biotechnology companies that produce COVID-19 vaccines made from plasmid DNA.
  • Plasmid DNA Contract Manufacturing Market Variables, Trends & Scope

First CureDuchenne Clinic Opens in Greater Dallas Area to Provide Specialized Care for Underserved Duchenne Muscular Dystrophy Patients

Retrieved on: 
Friday, March 3, 2023
Disease, Myasthenia gravis, Child, Health insurance coverage in the United States, SMA, Patient, Cardiology, Charcot–Marie–Tooth disease, Insurance, Duchenne muscular dystrophy, Education, Hope, Research, Neurology, Caregiver, Health, DMD, Private practice, Diagnosis, Spinal muscular atrophy, Becker muscular dystrophy, CMT, Nursing, Becker, Duchenne, Medicaid

DENTON, Texas, March 3, 2023 /PRNewswire/ -- CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, today announced the opening of The CureDuchenne Clinic, providing dedicated care and services to underserved and uninsured individuals with Duchenne and Becker muscular dystrophy. Located at the Neurology & Neuromuscular Care Center in Denton, Texas, the clinic will remove significant barriers of access for patients requiring highly specialized care, filling a gap in existing care for patients with the fatal neuromuscular disease.

Key Points: 
  • Located at the Neurology & Neuromuscular Care Center in Denton, Texas, the clinic will remove significant barriers of access for patients requiring highly specialized care, filling a gap in existing care for patients with the fatal neuromuscular disease.
  • The goal of the CureDuchenne Clinic is to provide high quality care for patients living with Duchenne or Becker muscular dystrophy from childhood through adulthood and to never deny care due to lack of insurance.
  • Duchenne muscular dystrophy is a progressive, muscle-wasting disease in children, affecting 15,000 individuals in the United States.
  • CureDuchenne was founded by Debra and Paul Miller in 2003 after their son was diagnosed with Duchenne muscular dystrophy.

Biohaven's Taldefgrobep Alfa Receives FDA Fast Track Designation for Spinal Muscular Atrophy

Retrieved on: 
Tuesday, February 21, 2023
Muscle atrophy, ALS, Myostatin, Muscle weakness, DSM-IV codes, NYSE, Efficacy, Patient, Drug, Facial muscles, Spinal muscular atrophy, Food, Orphan, Death, Safety, Disease, FDA, SMA, Growth, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Quality of life, Fast Track, Pharmaceutical industry, Dietary supplement

NEW HAVEN, Conn., Feb. 21, 2023 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN; "Biohaven") announced today that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for taldefgrobep alfa, a novel anti-myostatin adnectin, for the treatment of spinal muscular atrophy (SMA). Fast Track designation enables important new drugs to reach patients earlier by facilitating more frequent communications with the FDA and expeditious review of a drug which treats a serious condition and fills an unmet medical need. Biohaven previously received orphan drug designation from the FDA for taldefgrobep in the treatment of SMA.

Key Points: 
  • -  Taldefgrobep alfa, a myostatin-targeting biologic investigational agent, in Phase 3 development to increase muscle mass for Spinal Muscular Atrophy patients now granted Fast Track in addition to previously receiving Orphan Drug Designation in the US
    NEW HAVEN, Conn., Feb. 21, 2023 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN; "Biohaven") announced today that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for taldefgrobep alfa, a novel anti-myostatin adnectin, for the treatment of spinal muscular atrophy (SMA).
  • Biohaven previously received orphan drug designation from the FDA for taldefgrobep in the treatment of SMA.
  • Lindsey Lee Lair, M.D., M.B.A., F.A.A.N, Vice President, Clinical Development, Biohaven commented, "We are very pleased the FDA granted Fast Track designation for taldefgrobep alfa for the treatment of SMA.
  • We are excited about the potential for taldefgrobep alfa to improve the lives of patients and families affected by SMA."