Myopathy

Cabaletta Bio Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, March 21, 2024
Research, MPV, IIM, MuSK protein, Toxicity, Conference, Disease, Dermatomyositis, Juvenile dermatomyositis, Federation, Patient, NOL, Observation, CRS, SLE, Health Canada, IND, Immunoglobulin therapy, Pemphigus, Investment, Investigational New Drug, Myopathy, Systemic scleroderma, Marketing, FDA, Clinical trial, Cytokine release syndrome, Cyclophosphamide, CD19, Food, Autoimmune disease, Pharmaceutical industry

In October 2023, Cabaletta received Investigational New Drug (IND) application clearance from the U.S. Food and Drug Administration (FDA) for the Phase 1/2 RESET-SSc trial.

Key Points: 
  • In October 2023, Cabaletta received Investigational New Drug (IND) application clearance from the U.S. Food and Drug Administration (FDA) for the Phase 1/2 RESET-SSc trial.
  • In November 2023, Cabaletta announced that its IND application for CABA-201 was allowed to proceed by the FDA for the Phase 1/2 RESET-MG trial.
  • Cabaletta anticipates reporting initial clinical data from the Phase 1/2 RESET-MG trial in the second half of 2024.
  • As of December 31, 2023, Cabaletta had cash, cash equivalents and short-term investments of $241.2 million, compared to $106.5 million as of December 31, 2022.

MetrioPharm Establishes Scientific Advisory Board of Seasoned Immunology and Pediatric Experts

Retrieved on: 
Wednesday, March 13, 2024
Patient, Child, Laboratory, Duchenne muscular dystrophy, University of Oxford, School, Research, Inflammation, Electrophysiology, National Science Foundation, Immunology, Rockefeller University, Research fellow, Neuromuscular disease, Pediatrics, Pathology, SAB, IRB, CNRS, Neurology, Inflammation Research, Surgery, University, Neuro, MD, Senior, Consultant, Chemokine, Medical school, Retirement, CSO, Myopathy, Signal transduction, Doctor of Philosophy, Nursing

Ferdinando Nicoletti, MD, PhD, has been Full Professor of General Pathology and Immunology since 2011 at the University of Catania (Italy).

Key Points: 
  • Ferdinando Nicoletti, MD, PhD, has been Full Professor of General Pathology and Immunology since 2011 at the University of Catania (Italy).
  • “We welcome the members of MetrioPharm’s newly established scientific advisory board,” said Thomas Christély, CEO of MetrioPharm.
  • “We are happy to now formally establish and introduce this panel of experts for an even closer collaboration.
  • Every aspect of our research development program - from preclinical research to clinical trials - will benefit from the unparalleled expertise of our new scientific advisors.”

Study shows improved hand function using Carbonhand

Retrieved on: 
Wednesday, March 13, 2024
Jamar, Conference, Face, Perception, IBM, Glass, Facial muscles, Person, Myopathy, Pain, TMA, Hand, Autoimmune disease, Personal protective equipment

STOCKHOLM, March 13, 2024 /PRNewswire/ -- Today, a study on Improved physical function using a power-enhancing glove in persons with Inclusion Body Myositis was presented at the Global Conference on Myositis in Pittsburgh, USA.

Key Points: 
  • STOCKHOLM, March 13, 2024 /PRNewswire/ -- Today, a study on Improved physical function using a power-enhancing glove in persons with Inclusion Body Myositis was presented at the Global Conference on Myositis in Pittsburgh, USA.
  • The preliminary results show increased hand function and improved physical function for people with impaired hand function during use of Carbonhand.
  • The study was open to people with idiopathic inflammatory myopathies who perceived hand weakness.
  • The study included 40 persons with IBM that had reduced grip strength (kg) (md;range) (3.75;1.3-10.7) and physical function (IBM-FRS 20;2-38, IBM-PRO 23;1-44).

IGM Biosciences Announces Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, March 7, 2024
CD20, Progression-free survival, PFS, Myopathy, Research, Immunoglobulin M, Investment, FDA, Research and development, IGM, FOLFIRI, Myositis, Bevacizumab, Lupus, GAAP, Rheumatoid arthritis, Administration, Patient, Clinical trial, Survival, CD38, SLE, G&A, IND, Colorectal cancer, Pharmaceutical industry

MOUNTAIN VIEW, Calif., March 07, 2024 (GLOBE NEWSWIRE) -- IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company creating and developing engineered IgM antibodies, today announced its financial results for the fourth quarter and full year ended December 31, 2023 and provided an update on recent developments.

Key Points: 
  • MOUNTAIN VIEW, Calif., March 07, 2024 (GLOBE NEWSWIRE) -- IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company creating and developing engineered IgM antibodies, today announced its financial results for the fourth quarter and full year ended December 31, 2023 and provided an update on recent developments.
  • Collaboration Revenue: For the fourth quarter and year ended 2023, collaboration revenues were $0.7 million and $2.1 million, respectively, compared to $0.4 million and $1.1 million for the fourth quarter and year ended 2022, respectively.
  • Research and Development (R&D) Expenses: For the fourth quarter and year ended 2023, R&D expenses were $54.2 million and $215.5 million, respectively, compared to $45.0 million and $179.3 million for the fourth quarter and year ended 2022, respectively.
  • General and Administrative (G&A) Expenses: For the fourth quarter and year ended 2023, G&A expenses were $11.6 million and $50.1 million, respectively, compared to $11.6 million and $49.7 million for the fourth quarter and year ended 2022, respectively.

Satellos Presents Positive Preclinical Efficacy Data for SAT-3247 at the 2024 MDA Clinical & Scientific Conference

Retrieved on: 
Monday, March 4, 2024
Biotechnology, Health, Science, Pharmaceutical, Research, Molecule, OTCQB, Regeneration, MSCL, TSX, Animal, DMD, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy, Skeletal muscle, Conference, Event, FSHD, Poster, Trauma, Myopathy, Episcopal conference, Face

Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preclinical data showing SAT-3247 can improve skeletal muscle function in multiple mouse models of muscle degeneration.

Key Points: 
  • Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preclinical data showing SAT-3247 can improve skeletal muscle function in multiple mouse models of muscle degeneration.
  • In all instances, treatment with SAT-3247 over a three-to-four-week period resulted in a statistically significant improvement in muscle force versus animals receiving placebo.
  • We believe the muscle injury data further expand and broaden the potential for SAT-3247 into non-dystrophy disease indications as well as multiple forms of muscle injury or trauma in otherwise healthy individuals.
  • We continue on our development plan to advance SAT-3247 into first-in-human clinical trials mid-year.”
    These data are being presented in a poster at the 2024 MDA Clinical & Scientific Conference being held March 3-6 in Orlando.

Edgewise Therapeutics to Present on EDG-5506 for the Treatment of Becker Muscular Dystrophy at the 2024 MDA Clinical and Scientific Conference

Retrieved on: 
Wednesday, February 28, 2024
Biotechnology, Health, Pharmaceutical, Physical Therapy, Cardiology, BMD, Exercise, Principal investigator, Network, Myopathy, Edgewise, Conference, Muscular Dystrophy Association, University, Therapy, ARCH, Associate professor, University of Florida, Poster, Trade, Convention, MDA, Physiology, Episcopal conference, University of Copenhagen

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on EDG-5506, an investigational therapy designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies, at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on EDG-5506, an investigational therapy designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies, at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.
  • The conference will take place at the Hilton Orlando in Orlando, FL from March 3-6, 2024.
  • Posters will be showcased during the Networking & Poster Reception in the Exhibit Hall on Monday, March 4, 2024, from 6 – 8 PM ET.
  • The Edgewise presentation and posters will be available on the Edgewise website when they are presented.

Edgewise Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Thursday, February 22, 2024
Health, Other Health, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, Muscular dystrophy, Research, American Heart Association, Education, R9, MESA, Ageing, Fast Track, NSAA, McArdle, DUNE, Myopathy, Therapy, WMS, LYNX, ODD, Safety, Biomarker, Pharmacokinetics, HCM, Clinical trial, Caregiver, FOX, FDA, Muscle contraction, Engagement, Duchenne muscular dystrophy, ARCH, Administration, Patient, Disease, Pharmaceutical industry

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the fourth quarter and full year of 2023 and recent business highlights.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the fourth quarter and full year of 2023 and recent business highlights.
  • “In 2023, we made tremendous progress advancing our novel muscle-targeted therapeutics in the clinic,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise.
  • Research and development (R&D) expenses were $27.7 million for the fourth quarter of 2023, compared to $23.8 million for the immediately preceding quarter.
  • General and Administrative (G&A) expenses were $6.2 million for the fourth quarter of 2023, compared to $5.7 million for the immediately preceding quarter.

Avidity Biosciences Honors Rare Disease Day®

Retrieved on: 
Thursday, February 29, 2024
Rare disease, Therapy, SAN, Avidity, Caregiver, DMD, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy, Hope, Learning, DM1, RNA, Rare Disease Day, FSHD, Patient, Disease, AOC, Myopathy, Research

"Today, on Rare Disease Day, we are proud to join the global community in raising awareness for people living with rare diseases," said Sarah Boyce, president and chief executive officer at Avidity.

Key Points: 
  • "Today, on Rare Disease Day, we are proud to join the global community in raising awareness for people living with rare diseases," said Sarah Boyce, president and chief executive officer at Avidity.
  • "Recently, we had the privilege of hosting individuals affected by rare muscle diseases at Avidity.
  • Rare Disease Day takes place on the last day of February each year with the goal to raise awareness of the impact of rare diseases worldwide.
  • EURORDIS established Rare Disease Day in 2008 and coordinates with more than 70 national alliance patient organizations each year to honor those living with rare diseases as well as their families and caregivers.

New Research: Eating Almonds Can Aid in Post-Exercise Recovery

Retrieved on: 
Wednesday, February 21, 2024
Woman, Magnesium, Body composition, Senior lecturer, Overweight, Inflammation, Exercise, Muscle soreness, Potassium, Vitamin E, Frontiers, King's College London, Triglyceride, Nutrient, Fats, Creatine kinase, Athlete, C-reactive protein, Myopathy, Appetite, DV, Research, Public, Dietary supplement

MODESTO, Calif., Feb. 21, 2024 /PRNewswire/ -- A new study found that eating almonds reduced some feelings of muscle soreness during exercise recovery which translated to improved muscle performance during a vertical jump challenge. These results expand on prior research1 which looked at how almonds affect muscle recovery after exercise.

Key Points: 
  • These results expand on prior research1 which looked at how almonds affect muscle recovery after exercise.
  • The treadmill test was designed to cause muscle damage to see how almonds affected muscle recovery.
  • Study Conclusion: Snacking on almonds for eight weeks reduced perceived feelings of muscle soreness during recovery from muscle-damaging exercise, resulting in better maintenance of muscle functional capacity.
  • This study suggests that almonds are a functional food snack to improve exercise tolerance in mildly overweight, middle-aged adults.

Avidity Biosciences Receives FDA Rare Pediatric Disease Designation for AOC 1044 for Treatment of Duchenne Muscular Dystrophy in People with Mutations Amenable to Exon 44 Skipping

Retrieved on: 
Tuesday, February 20, 2024
Therapy, SAN, Avidity, PMOS, EMA, Orphan drug, Dystrophin, FDA, Food, DMD, Duchenne muscular dystrophy, Weakness, Safety, RNA, Skeletal muscle, European Medicines Agency, Pharmacokinetics, AOC, PMO, Science, Myopathy, Ageing, Pharmaceutical industry

In addition to receiving Rare Pediatric Disease Designation, AOC 1044 has been granted Orphan Designation by the FDA and the European Medicines Agency (EMA), and Fast Track Designation by the FDA.

Key Points: 
  • In addition to receiving Rare Pediatric Disease Designation, AOC 1044 has been granted Orphan Designation by the FDA and the European Medicines Agency (EMA), and Fast Track Designation by the FDA.
  • "We are pleased that the FDA has granted Rare Pediatric Disease designation to AOC 1044, adding to the Orphan Drug and Fast Track designations already granted.
  • Avidity plans to provide a first look at AOC 1044 data in people living with DMD44 in 2H 2024.
  • The EXPLORE44 trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial to evaluate AOC 1044 in healthy volunteers and participants with DMD mutations amenable to exon 44 skipping (DMD44).