Muscular Dystrophy Community Assistance Research and Education Amendments

Sarepta Therapeutics to Share Expression and Safety Results from Study 103 (ENDEAVOR) Evaluating SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

Retrieved on: 
Monday, May 17, 2021
Muscular dystrophy, Branches of biology, Dystrophin, Limb–girdle muscular dystrophy, Duchenne muscular dystrophy, Duchenne, DMD, Dystrophy, Muscular Dystrophy Community Assistance Research and Education Amendments, Exon skipping

Study 103 is the first clinical trial using Sarepta\xe2\x80\x99s commercially representative material for SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) for the treatment of Duchenne muscular dystrophy.

Key Points: 
  • Study 103 is the first clinical trial using Sarepta\xe2\x80\x99s commercially representative material for SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) for the treatment of Duchenne muscular dystrophy.
  • The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers.
  • We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development.
  • Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.

Global Limb-Girdle Muscular Dystrophy Markets, Epidemiology and Patient Flow Analysis Report 2021 - ResearchAndMarkets.com

Retrieved on: 
Friday, May 14, 2021
Health, General Health, Muscular dystrophy, Organ systems, Limb–girdle muscular dystrophy, Medicine, Dystrophy, Health, Muscular Dystrophy Community Assistance Research and Education Amendments, Limb girdle syndrome

b'The "Global Limb-Girdle Muscular Dystrophy Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nGlobal Limb-Girdle Muscular Dystrophy Epidemiology and Patient Flow Analysis - 2021, provides Limb-Girdle Muscular Dystrophy epidemiology, demographics, and patient flow.

Key Points: 
  • b'The "Global Limb-Girdle Muscular Dystrophy Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nGlobal Limb-Girdle Muscular Dystrophy Epidemiology and Patient Flow Analysis - 2021, provides Limb-Girdle Muscular Dystrophy epidemiology, demographics, and patient flow.
  • The data is presented by Pharma G7 countries including the US, France, Spain, Italy, UK, Japan.\nThe research provides population data to characterize Limb-Girdle Muscular Dystrophy patients, history of the disease at the population level (Limb-Girdle Muscular Dystrophy prevalence, Limb-Girdle Muscular Dystrophy incidence) and at the clinical level (from diagnosis to treated patients).
  • It also helps to identify patient sub-groups (age, gender, sub-groups) to understand targeted population for research and development, commercialization.\nLimb-Girdle Muscular Dystrophy patient flow: Limb-Girdle Muscular Dystrophy prevalence, diagnosed, and drug-treated patients\nDemographics: Limb-Girdle Muscular Dystrophy patients by age group, gender\nThe data from this research will help executives:\nEstablish basis for Limb-Girdle Muscular Dystrophy market sizing, assessing market potential, and developing drug forecast models\nIdentify Limb-Girdle Muscular Dystrophy patients segments through age groups, gender, and disease sub-types\nEvaluate Limb-Girdle Muscular Dystrophy market opportunities, identify target patient population\nView source version on businesswire.com: https://www.businesswire.com/news/home/20210514005192/en/\n'

Global Duchenne Muscular Dystrophy Epidemiology and Patient Flow Report 2021 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, May 5, 2021
Health, General Health, Muscular dystrophy, Organ systems, Dystrophy, Medicine, Duchenne muscular dystrophy, Duchenne, Branches of biology, Becker muscular dystrophy, Muscular Dystrophy Community Assistance Research and Education Amendments

b'The "Global Duchenne Muscular Dystrophy Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nGlobal Duchenne Muscular Dystrophy Epidemiology and Patient Flow Analysis - 2021, provides Duchenne Muscular Dystrophy epidemiology, demographics, and patient flow.

Key Points: 
  • b'The "Global Duchenne Muscular Dystrophy Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nGlobal Duchenne Muscular Dystrophy Epidemiology and Patient Flow Analysis - 2021, provides Duchenne Muscular Dystrophy epidemiology, demographics, and patient flow.
  • The data is presented by Pharma G7 countries including the US, France, Spain, Italy, UK, Japan.\nThe research provides population data to characterize Duchenne Muscular Dystrophy patients, history of the disease at the population level (Duchenne Muscular Dystrophy prevalence, Duchenne Muscular Dystrophy incidence) and at the clinical level (from diagnosis to treated patients).
  • It also helps to identify patient sub-groups (age, gender, sub-groups) to understand targeted population for research and development, commercialization.\nDuchenne Muscular Dystrophy patient flow: Duchenne Muscular Dystrophy prevalence, diagnosed, and drug-treated patients\nDemographics: Duchenne Muscular Dystrophy patients by age group, gender\nThe data from this research will help executives:\nEstablish basis for Duchenne Muscular Dystrophy market sizing, assessing market potential, and developing drug forecast models\nIdentify Duchenne Muscular Dystrophy patients segments through age groups, gender, and disease sub-types\nEvaluate Duchenne Muscular Dystrophy market opportunities, identify target patient population\nView source version on businesswire.com: https://www.businesswire.com/news/home/20210505005477/en/\n'

Sarepta Therapeutics to Present at Upcoming Investor Conferences

Retrieved on: 
Tuesday, May 4, 2021
Muscular dystrophy, Branches of biology, Dystrophin, Duchenne muscular dystrophy, Limb–girdle muscular dystrophy, Duchenne, DMD, Clinical medicine, Medical specialties, Muscular Dystrophy Community Assistance Research and Education Amendments

We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development.

Key Points: 
  • We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development.
  • Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.
  • For more information, please visit www.sarepta.com or follow us on Twitter , LinkedIn , Instagram and Facebook .\nWe routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com .
  • We encourage investors and potential investors to consult our website regularly for important information about us.\n"

Sarepta Therapeutics to Share Clinical Update for 30 mg/kg arm of MOMENTUM Study for SRP-5051, Its Investigational PPMO for the Treatment of Duchenne Muscular Dystrophy

Retrieved on: 
Friday, April 30, 2021
Muscular dystrophy, Branches of biology, Dystrophin, Limb–girdle muscular dystrophy, Duchenne muscular dystrophy, Duchenne, Clinical medicine, Anatomy, Muscular Dystrophy Community Assistance Research and Education Amendments

Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

Key Points: 
  • Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.
  • The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers.
  • We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development.
  • Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.

Parent Project Muscular Dystrophy Awards $450,000 to Collaborative Duchenne Muscular Dystrophy Care Projects

Retrieved on: 
Tuesday, April 6, 2021
Muscular dystrophy, Duchenne, Dystrophy, Nationwide Children's Hospital, Medicine, Branches of biology, Health, Biostrophin, Muscular Dystrophy Community Assistance Research and Education Amendments

HACKENSACK, N.J., April 6, 2021 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) is pleased to announce awards to three collaborative Certified Duchenne Care Center (CDCC) network projects totaling $450,000.

Key Points: 
  • HACKENSACK, N.J., April 6, 2021 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) is pleased to announce awards to three collaborative Certified Duchenne Care Center (CDCC) network projects totaling $450,000.
  • Her project will take place in partnership with the Ohio Pediatric Palliative and End-of-Life Care Network (OPPEN), which includes Nationwide Children's Hospital and Akron Children's Hospital, both Certified Duchenne Care Centers.
  • PPMD's Certified Duchenne Care Center Program supports standardized, comprehensive care and services for all people living with Duchenne.
  • Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.

Sarepta Therapeutics to Present Results from its Gene Therapy and RNA Platforms at the 2021 Annual MDA Clinical and Scientific Conference

Retrieved on: 
Monday, March 15, 2021
Muscular dystrophy, Branches of biology, Biology, Neurological disorders, Limb–girdle muscular dystrophy, Charcot–Marie–Tooth disease, Duchenne muscular dystrophy, Dystrophin, Dystrophy, Gene therapy, Muscular Dystrophy Community Assistance Research and Education Amendments, Sarepta Therapeutics

At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease.

Key Points: 
  • At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease.
  • The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development.
  • The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing.
  • For more information, please visit www.sarepta.com or follow us on Twitter , LinkedIn , Instagram and Facebook .

ReveraGen Receives $3.3 Million NIH Commercialization Readiness Pilot Grant for NDA Preparations for Vamorolone in Duchenne Muscular Dystrophy

Retrieved on: 
Tuesday, November 17, 2020
Organ systems, Branches of biology, Muscular dystrophy, Dystrophy, Becker muscular dystrophy, Vamorolone, Duchenne, Dystrophin, Small Business Innovation Research, Biostrophin, Muscular Dystrophy Community Assistance Research and Education Amendments

The Commercialization Readiness Pilot (CRP) program is designed to aid companies with previously funded SBIR/STTR Phase II/IIB projects as they advance to commercialization.

Key Points: 
  • The Commercialization Readiness Pilot (CRP) program is designed to aid companies with previously funded SBIR/STTR Phase II/IIB projects as they advance to commercialization.
  • A subsequent NIH SBIR Phase IIB award has supported the ongoing pivotal trial in 121 DMD boys recruited at 30 sites in 11 countries.
  • ReveraGen has open INDs for development of vamorolone in both Duchenne muscular dystrophy and Becker muscular dystrophy.
  • ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders.

Parent Project Muscular Dystrophy's Ground-Breaking Effort: An Update After One Year of Newborn Screening for Duchenne Muscular Dystrophy

Retrieved on: 
Tuesday, November 10, 2020
Muscular dystrophy, Medicine, Neonatology, Health, Epidemiology, Newborn screening, Duchenne, Dystrophy, Muscular Dystrophy Community Assistance Research and Education Amendments

HACKENSACK, N.J., Nov. 10, 2020 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , announced that the organization's Newborn Screening Pilot has been successfully screening babies born in New York State for Duchenne for over a year.

Key Points: 
  • HACKENSACK, N.J., Nov. 10, 2020 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , announced that the organization's Newborn Screening Pilot has been successfully screening babies born in New York State for Duchenne for over a year.
  • Data were presented at the Association of Public Health Laboratories Newborn Screening Virtual Symposium about the ongoing pilot in New York State.
  • It is the goal of PPMD and the Duchenne community to help eliminate the diagnostic delay in Duchenne through a public health program that includes newborn screening.
  • In addition to launching the Duchenne newborn screening pilot program, PPMD's newborn screening agenda includes active involvement on the reauthorization of the Newborn Screening Saves Lives Act ; annual Duchenne-specific language within Appropriations and Report Language to ensure federal partners are focused on Duchenne newborn screening efforts; engagement with the federal Advisory Committee on Heritable Disorders for Newborns and Children ; and leading the National Duchenne Newborn Screening Initiative, which has included the development of published care standards for newborns, ethical considerations for Duchenne newborn screening, and the publication of A Roadmap to Newborn Screening for Duchenne Muscular Dystrophy .

Dyne Therapeutics Announces Closing of Initial Public Offering

Retrieved on: 
Monday, September 21, 2020
Organ systems, Neurological disorders, RTT, Musculoskeletal disorders, Muscular dystrophy, Dystrophy, Facioscapulohumeral muscular dystrophy, Myotonic dystrophy, Dystrophin, Myotonia, Duchenne muscular dystrophy, Muscular Dystrophy Community Assistance Research and Education Amendments

WALTHAM, Mass., Sept. 21, 2020 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for patients with genetically driven diseases, today announced the closing of its initial public offering of 14,089,314 shares of its common stock at an initial public offering price of$19.00per share, including 1,837,736 additional shares of common stock issued upon the exercise in full by the underwriters of their option to purchase additional shares.

Key Points: 
  • WALTHAM, Mass., Sept. 21, 2020 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for patients with genetically driven diseases, today announced the closing of its initial public offering of 14,089,314 shares of its common stock at an initial public offering price of$19.00per share, including 1,837,736 additional shares of common stock issued upon the exercise in full by the underwriters of their option to purchase additional shares.
  • The gross proceeds of the offering, before deducting the underwriting discount and commissions and other offering expenses payable byDyne, were approximately$268 million.
  • Dyne Therapeutics is building a leading muscle disease company focused on advancing innovative life-transforming therapeutics for patients with genetically driven diseases.
  • Dyne is developing a broad portfolio of therapeutics for muscle diseases, including lead programs in myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD).