Muscular Dystrophy Community Assistance Research and Education Amendments

Long-term functional data from Sarepta Therapeutics’ Most Advanced Gene Therapy Programs to be Presented at Upcoming Annual Congress of the World Muscle Society

Retrieved on: 
Monday, September 14, 2020
Branches of biology, Health, Medicine, Muscular dystrophy, Becker muscular dystrophy, Duchenne muscular dystrophy, Limb–girdle muscular dystrophy, Dystrophy, Duchenne, Dystrophin, Muscular Dystrophy Community Assistance Research and Education Amendments, Sarepta Therapeutics

Casimersen Treatment in Eligible Patients with Duchenne Muscular Dystrophy: Safety, Tolerability, and Pharmacokinetics Over 144 Weeks of Treatment

Key Points: 
  • Casimersen Treatment in Eligible Patients with Duchenne Muscular Dystrophy: Safety, Tolerability, and Pharmacokinetics Over 144 Weeks of Treatment
    Open-Label Evaluation of Eteplirsen in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial
    Delay in Duchenne Muscular Dystrophy Progression with Eteplirsen: Attenuation of Pulmonary Decline and Projected Freedom from Continuous Ventilation
    Natural history and other presentations:
    Development of Cardiomyopathy, Respiratory Insufficiency and Loss of Ambulation in Becker Muscular Dystrophy: A Systematic Literature Review
    Disease Attributes Most Important from a Societal Perspective: A Case Study Involving Duchenne Muscular Dystrophy
    The Age at Loss of Ambulation Among Patients with Limb-Girdle Muscular Dystrophy (LGMD) Subtype 2: A Systematic Review
    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 .

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

Retrieved on: 
Wednesday, July 29, 2020
Muscular dystrophy, RTT, Organ systems, Duchenne muscular dystrophy, Duchenne, Dystrophy, Medicine, Neuromuscular disease, Biostrophin, Muscular Dystrophy Community Assistance Research and Education Amendments

HACKENSACK,N.J., July 29, 2020 /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 Program (CDCCP) with the certification of the clinic at Akron Children's Hospital (Akron Children's) in Akron, Ohio.

Key Points: 
  • HACKENSACK,N.J., July 29, 2020 /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 Program (CDCCP) with the certification of the clinic at Akron Children's Hospital (Akron Children's) in Akron, Ohio.
  • Akron Children's Neuromuscular program is a comprehensive multidisciplinary program designed to meet the unique needs of patients living with Duchenne muscular dystrophy and other neuromuscular disorders.
  • "We are thrilled to partner with Parent Project Muscular Dystrophy in our ongoing commitment to provide high quality care to our patients living with Duchenne.
  • PPMD's Certified Duchenne Care Center Program supports standardized, comprehensive care and services for all people living with Duchenne.

Catabasis Pharmaceuticals to Present at Parent Project Muscular Dystrophy Virtual Annual Conference

Retrieved on: 
Wednesday, July 15, 2020
Biotechnology, Health, Genetics, Pharmaceutical, Clinical trials, RTT, Catabasis Pharmaceuticals, Medicine, Dystrophy, Duchenne, Health, Muscular dystrophy, DMD, Branches of biology, Draft:CureDuchenne, Muscular Dystrophy Community Assistance Research and Education Amendments, Edasalonexent (CAT-1004), Catabasis

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it will present information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) at the Parent Project Muscular Dystrophy (PPMD) Virtual Annual Conference, which begins Wednesday, July 22, 2020.

Key Points: 
  • Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it will present information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) at the Parent Project Muscular Dystrophy (PPMD) Virtual Annual Conference, which begins Wednesday, July 22, 2020.
  • Additionally, Catabasis will share three posters during the Virtual Poster Session, which will also be available in the On-Demand Library.
  • Characteristics of Patients Who Enrolled in PolarisDMD, a Phase 3 Trial of Edasalonexent for Duchenne Muscular Dystrophy
    The On-Demand Library and the live stream will be made available to registered conference attendees.
  • Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy.

Parent Project Muscular Dystrophy Announces Pediatric Certified Duchenne Care Center, Children's Health and UT Southwestern Medical Center

Retrieved on: 
Wednesday, May 27, 2020
RTT, Duchenne, University of Texas Southwestern Medical Center, Dystrophy, Muscular dystrophy, Medicine, Branches of biology, Health, Muscular Dystrophy Community Assistance Research and Education Amendments

HACKENSACK, N.J., May 27, 2020 /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 Program (CDCCP) with the certification of the clinic at Children's Health in Dallas and UT Southwestern Medical Center (Children's Health and UT Southwestern).

Key Points: 
  • HACKENSACK, N.J., May 27, 2020 /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 Program (CDCCP) with the certification of the clinic at Children's Health in Dallas and UT Southwestern Medical Center (Children's Health and UT Southwestern).
  • Children's Health and UT Southwestern join the network as the 29th Certified Duchenne Care Center.
  • PPMD is thrilled to include Children's Health and UT Southwestern in their growing network of 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 Announces First Dual Pediatric & Adult Certified Duchenne Care Center, Kennedy Krieger Institute

Retrieved on: 
Wednesday, April 1, 2020
RTT, Muscular dystrophy, Duchenne, Dystrophy, Kennedy Krieger Institute, Health, Medicine, Articles, Muscular Dystrophy Community Assistance Research and Education Amendments

HACKENSACK, N.J., April 1, 2020 /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 Program (CDCCP) with the program's second Adult Certified Duchenne Care Center and first dually-certified center, the clinic at Kennedy Krieger Institute .

Key Points: 
  • HACKENSACK, N.J., April 1, 2020 /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 Program (CDCCP) with the program's second Adult Certified Duchenne Care Center and first dually-certified center, the clinic at Kennedy Krieger Institute .
  • While some of these certified centers are able to offer at least some aspects of care to adults with Duchenne, Kennedy Krieger is the first center to obtain certification in both pediatric and adult care.
  • Originally certified by PPMD in 2014, Kennedy Krieger Institute was the first center to seek dual pediatric and adult CDCC certification.
  • PPMD's Certified Duchenne Care Center Program supports standardized, comprehensive care and services for all people living with Duchenne.

Muscular Dystrophy Association to Host MDA Frontline COVID-19 Response with Dr. Barry Byrne, a Facebook Live Q&A on Protecting Neuromuscular Disease Community in Midst of COVID-19 Pandemic

Retrieved on: 
Tuesday, March 31, 2020
Neurological disorders, Organ systems, Amyotrophic lateral sclerosis, Muscular Dystrophy Association, Neuromuscular disease, Muscular dystrophy, Dystrophy, Health, Duchenne, Muscular Dystrophy Community Assistance Research and Education Amendments, Muscular Dystrophy Canada

The neuromuscular disease community people with ALS, muscular dystrophy, SMA, Duchenne and related rare diseases are among the highest risk populations for COVID-19.

Key Points: 
  • The neuromuscular disease community people with ALS, muscular dystrophy, SMA, Duchenne and related rare diseases are among the highest risk populations for COVID-19.
  • MDA will request questions from the community in forthcoming posts on the @MDAorg Facebook page.
  • ** panelists available for interview prior to, or following, Q&A**
    Dr. Barry J. Byrne, M.D., Ph.D: Dr. Byrne is a leading voice in neuromuscular disease research and care.
  • Since 1950, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people affected by muscular dystrophy, ALS and related neuromuscular diseases.

Invitae and Muscular Dystrophy Association (MDA) Expand Access to No-Charge Genetic Testing in the U.S. and Canada

Retrieved on: 
Monday, March 23, 2020
Muscular dystrophy, Medicine, Organ systems, Branches of biology, Becker muscular dystrophy, Duchenne muscular dystrophy, Genetic testing, Dystrophy, Muscular Dystrophy Association, Muscular Dystrophy Community Assistance Research and Education Amendments, Draft:CureDuchenne

Genetic testing can help accelerate diagnosis and treatment of conditions such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy which enables clinicians to begin identifying treatment options sooner," said Lynn O'Connor Vos, President and CEO of Muscular Dystrophy Association.

Key Points: 
  • Genetic testing can help accelerate diagnosis and treatment of conditions such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy which enables clinicians to begin identifying treatment options sooner," said Lynn O'Connor Vos, President and CEO of Muscular Dystrophy Association.
  • Detect Muscular Dystrophy also offers genetic testing to family members of patients with genetic variants associated with disease to better understand their own disease risks.
  • "We're proud to work with MDA to increase access to early genetic testing among patients suspected of having muscular dystrophy.
  • Invitae sponsored testing programs are designed to increase access to genetic testing, particularly in conditions where earlier testing can improve diagnosis and treatment yet testing remains underutilized.

Nonprofit CureDuchenne Launches Biobank to Fuel Research for Duchenne Muscular Dystrophy

Retrieved on: 
Saturday, October 12, 2019
Biotechnology, Pharmaceutical, Health, Muscular dystrophy, RTT, Duchenne, Medicine, Dystrophy, Branches of biology, Health, Draft:CureDuchenne, Muscular Dystrophy Community Assistance Research and Education Amendments

CureDuchenne, a global leader in research, patient care, and innovation in improving and extending the lives of those living with Duchenne muscular dystrophy (Duchenne), announced today it has initiated the development of a biobank for Duchenne.

Key Points: 
  • CureDuchenne, a global leader in research, patient care, and innovation in improving and extending the lives of those living with Duchenne muscular dystrophy (Duchenne), announced today it has initiated the development of a biobank for Duchenne.
  • The CureDuchenne Biobank is dedicated to empowering Duchenne research and the search for a cure for Duchenne that is independent and accessible to all researchers.
  • To fill that gap, its important to provide researchers with free and unrestricted access to the cell lines they need for Duchenne research, Miller added.
  • CureDuchenne is the nations leading nonprofit organization dedicated to finding a cure for Duchenne, the most common and lethal form of muscular dystrophy.

CureDuchenne Announces 2020 Line-Up of Educational Events Across U.S. for Duchenne Community

Retrieved on: 
Wednesday, September 18, 2019
Biotechnology, Family, Health, Consumer, Other Health, Philanthropy, Research, General Health, Foundation, Science, Muscular dystrophy, Duchenne, Dystrophy, Muscular Dystrophy Community Assistance Research and Education Amendments

CureDuchenne, a leading nonprofit in Duchenne research, patient care and innovation, announced today its line-up of CureDuchenne Cares events that will take place across the United States in 2020.

Key Points: 
  • CureDuchenne, a leading nonprofit in Duchenne research, patient care and innovation, announced today its line-up of CureDuchenne Cares events that will take place across the United States in 2020.
  • CureDuchenne Cares, a division of CureDuchenne, is an interactive and educational outreach program for patients, families and health care professionals in the Duchenne muscular dystrophy community.
  • A complete listing of CureDuchenne Cares 2020 dates and locations can be found here: https://www.cureduchenne.org/cureduchenne-cares-2020-events/ .
  • Entirely underwritten by sponsors and provided at no cost to the Duchenne community, the CureDuchenne Cares program hosts three different formats of educational events Sessions, Workshops and FUTURES.

Sarepta Therapeutics Announces Recipients of Route 79, The Duchenne Scholarship Program

Retrieved on: 
Thursday, September 5, 2019
Muscular dystrophy, RTT, Duchenne, Sarepta Therapeutics, Life sciences, Dystrophy, Articles, Medicine, Biotechnology, Sulfones, Draft:CureDuchenne, Muscular Dystrophy Community Assistance Research and Education Amendments

CAMBRIDGE, Mass., Sept. 05, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the recipients of Route 79, The Duchenne Scholarship Program.

Key Points: 
  • CAMBRIDGE, Mass., Sept. 05, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the recipients of Route 79, The Duchenne Scholarship Program.
  • On behalf of Sarepta and members of the selection committee, it is our great privilege to announce the recipients of Route 79, The Duchenne Scholarship Program.
  • 2019 Named Recipients - Route 79, The Duchenne Scholarship Program
    Scholarship recipients are chosen by an independent committee of Duchenne community members based on each applicants essay and demonstrated level of community involvement.
  • Route 79, TheDuchenne Scholarship Program is designed to help students diagnosed with Duchenne muscular dystrophy (Duchenne) pursue their post-high school educational goals.