Dystrophinopathy

Edgewise Receives Orphan Drug and Rare Pediatric Disease Designations for Its Muscular Dystrophy Program from FDA

Retrieved on: 
Thursday, November 30, 2023
Research, Neurology, FDA, Genetics, Clinical Trials, Biotechnology, General Health, Pharmaceutical, Health, Science, Becker muscular dystrophy, Therapy, ODD, Dystrophinopathy, Marketing, Duchenne muscular dystrophy, PDUFA, Fast Track, Pharmaceutical industry, Duchenne, Becker

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food & Drug Administration (FDA) has granted EDG-5506 Orphan Drug Designation (ODD) for the treatment of Duchenne muscular dystrophy (Duchenne) and Becker muscular dystrophy (Becker) and Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food & Drug Administration (FDA) has granted EDG-5506 Orphan Drug Designation (ODD) for the treatment of Duchenne muscular dystrophy (Duchenne) and Becker muscular dystrophy (Becker) and Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne.
  • EDG-5506 is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne and Becker.
  • The FDA previously granted Fast Track designation for the investigation and development of EDG-5506 for the treatment of Becker.
  • “Receiving orphan drug and rare pediatric disease designations are important milestones in advancing our novel small molecule therapeutic approach to treating individuals with Duchenne and Becker,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise.

Edgewise Therapeutics Reports Third Quarter 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Thursday, November 9, 2023
Biotechnology, Health, Pharmaceutical, Physical Therapy, Cardiology, LYNX, Research, FOX, DUNE, Ageing, Education, Biomarker, Muscular dystrophy, Muscular Dystrophy Association, NSAA, G&A, Myopathy, Glycogen storage disease type V, American Heart Association, WMS, R, Muscle contraction, Pharmacokinetics, Dystrophinopathy, Disorder, Glycogen, PK, Clinical trial, Duchenne muscular dystrophy, McArdle, Safety, HCM, Therapy, Patient, MESA, R9, Administration, Pharmaceutical industry, Edgewise, Duchenne, Becker

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the third quarter 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 third quarter of 2023 and recent business highlights.
  • “I’m thrilled with the progress we’ve made in 2023 across our skeletal and cardiovascular programs,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise.
  • Research and development (R&D) expenses were $23.8 million for the third quarter of 2023, compared to $19.6 million for the immediately preceding quarter.
  • General and Administrative (G&A) expenses were $5.7 million for the third quarter of 2023, compared to $5.8 million for the immediately preceding quarter.

Edgewise Therapeutics Announces Expansion of their EDG-5506 Clinical Program in Duchenne Muscular Dystrophy (Duchenne)

Retrieved on: 
Thursday, October 26, 2023
Research, Pharmaceutical, People with Disabilities, Consumer, Genetics, Teens, Children, Clinical Trials, Science, Biotechnology, FDA, Health, Steroid, LYNX, FOX, Adolescence, Ageing, MDA, Biomarker, University of Florida, NSAA, Myopathy, Pharmacokinetics, Dystrophinopathy, PK, University, Safety, Therapy, Patient, Pharmaceutical industry, Edgewise, Duchenne

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced an expansion of their clinical development program of EDG-5506, an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced an expansion of their clinical development program of EDG-5506, an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne.
  • The Company is initiating FOX, a new Phase 2 placebo-controlled trial in children and adolescent boys with Duchenne who have been previously treated with gene therapy.
  • The FOX trial will assess the effect of EDG-5506 over 12 weeks on safety, pharmacokinetics (PK) and biomarkers of muscle damage.
  • The study will also explore changes in functional measures, such as the North Star Ambulatory Assessment (NSAA) and self-reported/caregiver-reported outcomes.

Edgewise Therapeutics to Present on EDG-5506 for Becker Muscular Dystrophy at the 28th International Annual Congress of the World Muscle Society

Retrieved on: 
Wednesday, September 27, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Cardiology, Dystrophinopathy, WMS, Muscular dystrophy, Becker muscular dystrophy, Drug, Trial of the century, ARCH, Congress, Edgewise, Natural history, Data, Therapy, Pharmaceutical industry, Becker

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the Company will present on EDG-5506, an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, at the 28th International Annual Congress of the World Muscle Society (WMS).

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the Company will present on EDG-5506, an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, at the 28th International Annual Congress of the World Muscle Society (WMS).
  • The conference will take place at the Charleston Convention Center in Charleston, South Carolina, from October 3-7, 2023.
  • Details of the Edgewise symposium and scientific posters at WMS:
    On Tuesday, October 3, 2023, at 4:30 pm ET, Edgewise will sponsor a symposium, “Becker Muscular Dystrophy Natural History and ARCH, an Open Label Study in Becker: Putting the Data in Context.” The symposium will feature presentations by Edgewise leadership and leading neuromuscular disease experts:
    Sam Collins, M.D., Ph.D., Vice President of Clinical Development, Edgewise Therapeutics
    Only registered conference attendees can register for the symposium.
  • Title: Characterization of short- and long-term proteomic response to the fast skeletal myosin inhibitor, EDG-5506, in Becker muscular dystrophy (P.254)
    The Edgewise symposium presentation and posters will be available on the Edgewise website when they are presented.

Edgewise Therapeutics Announces Initiation of GRAND CANYON, a Global Pivotal Study of EDG-5506 in Becker Muscular Dystrophy (Becker)

Retrieved on: 
Tuesday, September 26, 2023
Health, Physical Therapy, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, Dystrophinopathy, Creatine kinase, Myopathy, Duchenne muscular dystrophy, Muscular dystrophy, Pharmacokinetics, Biomarker, Duchenne, NSAA, ARCH, PPMD, Entrepreneurship, Ageing, Therapy, Safety, Dietary supplement, Becker, Edgewise

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced the start of enrollment of GRAND CANYON, a global pivotal study of EDG-5506 in individuals with Becker.

Key Points: 
  • Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced the start of enrollment of GRAND CANYON, a global pivotal study of EDG-5506 in individuals with Becker.
  • EDG-5506 is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Becker and Duchenne muscular dystrophy.
  • There are currently no approved therapies for individuals with Becker, a serious genetic, progressive neuromuscular disorder with significant unmet need.
  • GRAND CANYON is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of EDG-5506 in adults with Becker.

Edgewise Therapeutics Announces Positive 12-Month Topline Results from the ARCH Open Label Study of EDG-5506 in Adults with Becker Muscular Dystrophy (BMD)

Retrieved on: 
Monday, June 26, 2023
Health, Genetics, Health Technology, Clinical Trials, Pharmaceutical, Biotechnology, Dystrophinopathy, Myopathy, Duchenne muscular dystrophy, Pharmacokinetics, Multimedia, Biomarker, Duchenne, BMD, NSAA, ARCH, DMD, Edgewise, TNNI2, CK, Therapy, Safety, Pharmaceutical industry

EDG-5506 is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including BMD and Duchenne muscular dystrophy (DMD).

Key Points: 
  • EDG-5506 is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including BMD and Duchenne muscular dystrophy (DMD).
  • The Company is reporting data at the end of 12 months of treatment with EDG-5506.
  • Further, Edgewise plans to discuss these data at the Parent Project Muscular Dystrophy Annual Conference, June 29 – July 1, 2023.
  • The Edgewise presentations will be available on the Edgewise website when they are presented.

Parent Project Muscular Dystrophy Announces Pediatric Certified Duchenne Care Center, Norton Children's Hospital

Retrieved on: 
Tuesday, June 20, 2023
Mischer Neuroscience Institute, Duchenne muscular dystrophy, University, MSN, Amanda Rogers, Clinical Care Classification System, Norton Children's Hospital, Duchenne, History, Associate, Muscular dystrophy, U.S. News & World Report, CDC, Certification, University of Louisville School of Medicine, DO, Health, CPN, Patient, PPMD, Dystrophinopathy, RN, News, Nursing, Becker, DRS

WASHINGTON, June 20, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced the expansion of its renowned Certified Duchenne Care Center (CDCC) Program with the certification of the clinic at Norton Children's in Louisville, KY. This is an exciting step for the CDCC Program as it brings standardized Duchenne care to a new region of the country with the first certification in the state of Kentucky.

Key Points: 
  • WASHINGTON, June 20, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , announced the expansion of its renowned Certified Duchenne Care Center (CDCC) Program with the certification of the clinic at Norton Children's in Louisville, KY.
  • PPMD's Certified Duchenne Care Center Program supports standardized, comprehensive care and services for all people living with Duchenne.
  • Mena Scavina, DO, PPMD's Certified Duchenne Care Center Program Advisor is delighted to add Norton Children's to the growing network of Certified Duchenne Care Centers and to expand the program to include its first center in the state of Kentucky.
  • Click here to learn more about the history of PPMD's Certified Duchenne Care Center Program and to access PPMD's first published article on the program.

Parent Project Muscular Dystrophy Announces Pediatric Certified Duchenne Care Center, Boston Children's Hospital

Retrieved on: 
Friday, June 16, 2023
Partnership, Boston Children's Hospital, Duchenne muscular dystrophy, Clinical Care Classification System, Boston, Duchenne, History, Associate, Certification, CDC, Health, DMD, Patient, PPMD, Dystrophinopathy, Hospital, Muscular dystrophy, Nursing, Becker

WASHINGTON, June 16, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced the expansion of its renowned Certified Duchenne Care Center (CDCC) Program with the certification at Boston Children's Hospital. This is an exciting step for the CDCC Program as it brings standardized Duchenne care to Massachusetts.

Key Points: 
  • WASHINGTON, June 16, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , announced the expansion of its renowned Certified Duchenne Care Center (CDCC) Program with the certification at Boston Children's Hospital .
  • "Boston Children's comprehensive neuromuscular clinic includes sub-specialists with expertise in muscular dystrophy to address the various needs of individuals with Duchenne and Becker.
  • "We at Boston Children's Hospital's multidisciplinary DMD center are really excited to be part of PPMD's Certified Duchenne Care Centers.
  • PPMD's Certified Duchenne Care Center Program supports standardized, comprehensive care and services for all people living with Duchenne.

Edgewise Therapeutics to Present on EDG-5506 for Becker and Duchenne Muscular Dystrophy (BMD, DMD) at the 2023 Annual MDA Clinical and Scientific Conference

Retrieved on: 
Tuesday, March 14, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Cardiology, Muscular Dystrophy Association, University, MBBS, Associate, Dystrophinopathy, Principal investigator, Drug, Hilton Anatole, Episcopal conference, National Primate Research Exhibition Hall, Conference, Office of Rare Diseases Research, Doctor of Philosophy, Pediatrics, Trade, Therapy, Poster, MDA, Pharmaceutical industry, Textile printing, Edgewise, Becker muscular dystrophy

The conference will take place at the Hilton Anatole in Dallas, TX from March 19-22, 2023.

Key Points: 
  • The conference will take place at the Hilton Anatole in Dallas, TX from March 19-22, 2023.
  • Posters will be viewable both in-person during the conference from March 19-21, 2023 and showcased in the Exhibit Halls from 6:00-8:00 pm CT.
  • The full MDA 2022 Conference program is available here: https://mdaconference.org .
  • The Edgewise presentation and posters will be available on the Edgewise website when they are presented.

Parent Project Muscular Dystrophy Announces Pediatric Certified Duchenne Care Center, Phoenix Children's

Retrieved on: 
Friday, February 17, 2023
PPMD, Phoenix Children's Hospital, Travel, Patient, Education, Certification, Barrow Neurological Institute, Child, Parent, Duchenne, APRN, CDC, History, Duchenne muscular dystrophy, MS, Nursing, Becker, Dystrophinopathy

WASHINGTON, Feb. 17, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced the expansion of their renowned Certified Duchenne Care Center (CDCC) Program with the certification of Phoenix Children's, one of the nation's fastest-growing pediatric healthcare systems. This addition to the CDCC Program is the first certification in the state of Arizona, and expands the network as the first center to capture the broader Duchenne and Becker population living in the Southwest.

Key Points: 
  • WASHINGTON, Feb. 17, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , announced the expansion of their renowned Certified Duchenne Care Center (CDCC) Program with the certification of Phoenix Children's , one of the nation's fastest-growing pediatric healthcare systems.
  • PPMD's Certified Duchenne Care Center Program supports standardized, comprehensive care and services for all people living with Duchenne.
  • As part of its ongoing mission to end Duchenne, PPMD continues to insist that all people with Duchenne receive comprehensive care.
  • Click here to learn more about the history of PPMD's Certified Duchenne Care Center Program and to access PPMD's first published article on the program.