Draft:CureDuchenne

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.

Solid Biosciences Announces Appointment of Ian F. Smith as Chairman of the Board of Directors

Retrieved on: 
Thursday, June 18, 2020
Muscular dystrophy, Duchenne, Dystrophy, Medicine, Health, Draft:CureDuchenne, Branches of biology

CAMBRIDGE, Mass., June 18, 2020 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy, announced today the appointment of board member Ian F. Smith as Chairman of the Board.

Key Points: 
  • CAMBRIDGE, Mass., June 18, 2020 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy, announced today the appointment of board member Ian F. Smith as Chairman of the Board.
  • Mr. Smith succeeds Andrey Zarur, Ph.D., who served as Chairman of the Board since 2013 and is one of the co-founders of Solid Biosciences.
  • He added, We are honored to have Ian lead the board in this next phase for Solid Biosciences.
  • Solid Biosciences is a life sciences company focused on developing transformative treatments to improve the lives of patients living with Duchenne muscular dystrophy (Duchenne).

Team Joseph Unites With Duchenne Muscular Dystrophy Community to Help Families During COVID-19

Retrieved on: 
Tuesday, June 16, 2020
Muscular dystrophy, RTT, Duchenne, Dystrophy, Medicine, Branches of biology, Health, Draft:CureDuchenne

That is why Team Joseph and ten other Duchenne nonprofits are working together to ease the additional challenges Duchenne families are currently facing.

Key Points: 
  • That is why Team Joseph and ten other Duchenne nonprofits are working together to ease the additional challenges Duchenne families are currently facing.
  • About 12,000 children and young adults are living with Duchenne, a progressive form of muscular dystrophy, in the United States.
  • "The situation we are facing with COVID-19 is unprecedented, but so is the collaboration we are seeing among Duchenne organizations," said Marissa Penrod, Team Joseph Founder/CEO.
  • Team Joseph is a non-profit organization whose mission is to aggressively fund cutting-edge research to find a treatment or cure for Duchenne muscular dystrophy.

Jesse's Journey grants $1M toward the launch of a clinical trial for Duchenne muscular dystrophy in Canada

Retrieved on: 
Wednesday, June 10, 2020
Muscular dystrophy, RTT, Organ systems, Health research, Jesse's Journey, Health, Medicine, Duchenne, Dystrophy, Vamorolone, Biostrophin, Draft:CureDuchenne

The funding from Jesse's Journey ensures that sites across Canada will be supported to recruit boys with Duchenne into the new study.

Key Points: 
  • The funding from Jesse's Journey ensures that sites across Canada will be supported to recruit boys with Duchenne into the new study.
  • "Families living with Duchenne across Canada and the academic medical centers have been amazing collaborators with the vamorolone development team.
  • Jesse's Journey is Canada's leading charity in the fight to defeat Duchenne muscular dystrophy.
  • ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders.

CureDuchenne Ventures Announces The "CureDuchenne Ventures 2020 Pitch Contest," To Award Two $25,000 Prizes For Novel Ideas In Duchenne Muscular Dystrophy Therapy Development

Retrieved on: 
Tuesday, June 9, 2020
RTT, Duchenne, Muscular dystrophy, Dystrophy, Medicine, Health, Draft:CureDuchenne, Branches of biology, Jesse's Journey

Our recent $1M seed investment into Myosana Therapeutics , exemplifies our commitment to funding and supporting next generation gene therapy research."

Key Points: 
  • Our recent $1M seed investment into Myosana Therapeutics , exemplifies our commitment to funding and supporting next generation gene therapy research."
  • For over a decade, CureDuchenne Ventures has funded more than 20 promising research projects resulting in six successful exits to advance treatments for Duchenne.
  • The CureDuchenne Ventures Pitch Contest creates a new platform that encourages and supports innovative ideas to accelerate research for the Duchenne community.
  • The two (2) eligible entrants with the most promising ideas in Duchenne Muscular Dystrophy therapy development will each win $25,000.

Duchenne UK and Parent Project Muscular Dystrophy Announce 2020 Joint Research Grant Call

Retrieved on: 
Tuesday, June 2, 2020
Muscular dystrophy, RTT, Duchenne, Dystrophy, Medicine, Branches of biology, Health, Draft:CureDuchenne, Biostrophin

HACKENSACK, N.J, June 2, 2020 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a US nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , and Duchenne UK , a UK-based patient organization, are pleased to announce the launch of their joint 2020 Research Grant Call, inviting research proposals for submission and review.

Key Points: 
  • HACKENSACK, N.J, June 2, 2020 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a US nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , and Duchenne UK , a UK-based patient organization, are pleased to announce the launch of their joint 2020 Research Grant Call, inviting research proposals for submission and review.
  • Up to $1 million (US) will be awarded to a project or projects seeking to minimize the immunological issues of gene therapy to treat Duchenne.
  • Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenneour mission is to end Duchenne.
  • Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy.

Solid Biosciences Presents Data from Duchenne Muscular Dystrophy Gene Therapy Program at ASGCT 23rd Annual Meeting

Retrieved on: 
Thursday, May 14, 2020
RTT, Muscular dystrophy, Duchenne muscular dystrophy, Branches of biology, Duchenne, Medicine, Dystrophy, Genetics, Draft:CureDuchenne, Biostrophin

CAMBRIDGE, Mass., May 14, 2020 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) today announced the presentation of clinical biomarker data from its SGT-001 microdystrophin gene therapy clinical trial for the treatment of Duchenne muscular dystrophy (Duchenne) on May 13 at the American Society of Gene and Cell Therapy (ASGCT) 23rd Annual Meeting, which is being held virtually.

Key Points: 
  • CAMBRIDGE, Mass., May 14, 2020 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) today announced the presentation of clinical biomarker data from its SGT-001 microdystrophin gene therapy clinical trial for the treatment of Duchenne muscular dystrophy (Duchenne) on May 13 at the American Society of Gene and Cell Therapy (ASGCT) 23rd Annual Meeting, which is being held virtually.
  • SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy.
  • Solids SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy (Duchenne).
  • Solid Biosciences is a life sciences company focused on developing transformative treatments to improve the lives of patients living with Duchenne muscular dystrophy (Duchenne).

Capricor Announces Positive Top-Line Final Results from HOPE-2 Study in Patients with Duchenne Muscular Dystrophy Treated with Lead Candidate CAP-1002

Retrieved on: 
Wednesday, May 13, 2020
RTT, Medicine, Priority review, Medical specialties, Duchenne muscular dystrophy, Duchenne, Muscular dystrophy, Dystrophy, Branches of biology, Draft:CureDuchenne

It has met our expectations in terms of clinical meaningfulness in this population of patients where treatment options are extremely limited.

Key Points: 
  • It has met our expectations in terms of clinical meaningfulness in this population of patients where treatment options are extremely limited.
  • With the exception of hypersensitivity reactions which were mitigated with a common pre-medication regimen, no safety signals were identified in the HOPE-2 trial.
  • If Capricor were to receive market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher.
  • Capricors lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy.

Catabasis Pharmaceuticals Presents Edasalonexent, a Potential Foundational Therapy for Duchenne Muscular Dystrophy

Retrieved on: 
Tuesday, May 12, 2020
Pharmaceutical, Health, Clinical trials, Catabasis Pharmaceuticals, RTT, Organ systems, Neurological disorders, Health, Dystrophy, Duchenne, Muscular Dystrophy Association, Muscular dystrophy, Draft:CureDuchenne, Rimeporide

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) in poster presentations at the 2020 Muscular Dystrophy Association (MDA) Virtual Poster Session.

Key Points: 
  • Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) in poster presentations at the 2020 Muscular Dystrophy Association (MDA) Virtual Poster Session.
  • As we advance our Phase 3 PolarisDMD trial, we gain valuable insights into the full potential of edasalonexent, said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis.
  • Those living with Duchenne have limited treatment options, and our goal is for edasalonexent to be a new foundational therapy.
  • Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy.

Global Casimersen Drug Insights, 2020-2030

Retrieved on: 
Thursday, April 30, 2020
RTT, Life sciences, Articles, Medicine, Biotechnology, Corvallis, Oregon, Sarepta Therapeutics, Duchenne muscular dystrophy, Dystrophy, Duchenne, Muscular dystrophy, Draft:CureDuchenne

Casimersen is being developed by Sarepta Therapeutics in phase II stage of development for the treatment of Duchenne Muscular Dystrophy.

Key Points: 
  • Casimersen is being developed by Sarepta Therapeutics in phase II stage of development for the treatment of Duchenne Muscular Dystrophy.
  • The report also highlights the drug research and development activity details across the United States, Europe and Japan.
  • A detailed description of regulatory milestones, development activities, and some key findings provide the current development scenario of Casimersen.
  • What are the other emerging products available and how these are giving competition to Casimersen?