Duchenne Muscular Dystrophy – Page 2

Photo courtesy of Elijah Stacey

Rare Community Profiles: Destroy Duchenne: How Elijah Stacy is Working to Improve the Lives of People with Duchenne Muscular Dystrophy

Post author:Jessica Lynn
Post published:November 9, 2023
Post category:Duchenne Muscular Dystrophy

Rare Community Profiles Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families,…

source: pixabay.com

Death of Muscular Dystrophy Patient Potentially Caused by Adeno-Associated Virus That Delivered Gene Therapy

Post author:Rose Duesterwald
Post published:October 13, 2023
Post category:Duchenne Muscular Dystrophy X-linked myotubular myopathy

For over four decades, the aspiration of gene therapy has been to develop novel therapies with the potential to enhance human health. Recombinant AAV (rAAV), a type of gene therapy,…

CureDuchenne Launches the CureDuchenne Caregiver Course to Support Caregivers of People with DMD

Post author:Jessica Lynn
Post published:September 28, 2023
Post category:Duchenne Muscular Dystrophy

Since its founding 20 years ago, CureDuchenne has been tirelessly working to find and fund a cure for Duchenne muscular dystrophy (DMD). They have done this through supporting families,…

source: pixabay.com

NS-089/NCNP-02 Nabs Orphan Drug Designation for DMD

Post author:Jessica Lynn
Post published:August 15, 2023
Post category:Duchenne Muscular Dystrophy

NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., recently announced via news release that its therapy NS-089/NCNP-02 was granted Orphan Drug designation from the U.S. Food and Drug…

SAT-3153 Earns Orphan Drug Designation for DMD

Post author:Jessica Lynn
Post published:August 6, 2023
Post category:Duchenne Muscular Dystrophy

Earlier this year, the FDA granted Accelerated Approval to ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatmnt of Duchenne muscular dystrophy (DMD) in boys aged 4-5. While this is a stunning…

source: shutterstock.com

These Clinical Trials Could be Game Changers in the Latter Half of 2023

Post author:James Moore
Post published:July 14, 2023
Post category:Duchenne Muscular Dystrophy Familial Hypercholesterolemia Heterozygous Familial Hypercholesterolemia Multiple Sclerosis Phase 3 clinical trial Phase III Trial Transthyretin amyloid cardiomyopathy

In a story from BioPharma Dive, a number of high-profile clinical trials are under way that could see conclusive results by the end of 2023. While some of these are…

source: shutterstock.com

A Top FDA Official Overrules Reviewers’ Rejection of Sarepta’s DMD Gene Therapy

Post author:Rose Duesterwald
Post published:July 12, 2023
Post category:Duchenne Muscular Dystrophy

It looked as if the fate of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy was pretty much sealed until an FDA official intervened. This information was provided by…

source: shutterstock.com

Rare Community Profiles: A Partnership Between CureDuchenne and PicnicHealth Bolsters the Power of CureDuchenne Link

Post author:Jessica Lynn
Post published:June 22, 2023
Post category:Duchenne Muscular Dystrophy

Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

source: pixabay.com

Duchenne Muscular Dystrophy Patient Dies in Gene Therapy Trial

Post author:Rose Duesterwald
Post published:June 1, 2023
Post category:Duchenne Muscular Dystrophy

Fierce Biotech recently carried an article about the death of Terry Horgan. Terry was a 27-year-old Duchenne muscular dystrophy (DMD) patient and the brother of the CEO of the…

Source: https://pixabay.com/en/sky-clouds-rays-sun-hope-sunbeam-1107952/

MP1032 Earns Orphan Drug Designation for DMD

Post author:Jessica Lynn
Post published:May 30, 2023
Post category:Duchenne Muscular Dystrophy Timely

As reported in Yahoo! Finance, the United States’ Food and Drug Administration (FDA) recently granted Orphan Drug designation to MP1032. This therapy, developed by clinical-stage biotech company MetrioPharm, is being…

Sarepta’s Duchenne Gene Therapy Drug Cleared for Accelerated Approval

Post author:Rose Duesterwald
Post published:May 16, 2023
Post category:Duchenne Muscular Dystrophy

Gene therapy has seemed out of reach for decades. Yet advocates have considered it a way to have damaged or missing genes restore dystrophin and other proteins. Doctors and advocates…

source: shutterstock.com

Rare Community Profiles: CureDuchenne Clinic in Greater Dallas Area Aims to Improve Treatment Accessibility and Care for Underserved Families with DMD and BMD

Post author:Jessica Lynn
Post published:May 10, 2023
Post category:Becker muscular dystrophy Duchenne Muscular Dystrophy

Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

source: pixabay.com

Teen with DMD Finishes 12th Flying Pig Marathon

Post author:Jessica Lynn
Post published:May 10, 2023
Post category:Duchenne Muscular Dystrophy

The citizens of Cincinnati know one universal truth: that the annual Flying Pig Marathon weekend will bring fun, family, and fulfillment to their city. And with that universal truth comes…

source: shutterstock.com

Experimental Duchenne Muscular Dystrophy Treatment Earns Fast Track Designation

Post author:James Moore
Post published:May 1, 2023
Post category:Duchenne Muscular Dystrophy

According to a story from PR Newswire, the biopharmaceutical company Avidity Biosciences, Inc., recently announced that its investigational therapy AOC 1044 has received the US Food and Drug Administration's Fast…

905513 / Pixabay

MDA Announces Research Grants for Duchenne Muscular Dystrophy

Post author:Jessica Lynn
Post published:April 4, 2023
Post category:Duchenne Muscular Dystrophy

Medical research is incredibly important, especially within the rare disease community. Rare conditions tend to be under-researched and under-funded; this can make it difficult not only to better learn…

PublicDomainPictures / Pixabay

FDA Grants Orphan Drug Designation to DYNE-251 for DMD

Post author:Jessica Lynn
Post published:April 4, 2023
Post category:Duchenne Muscular Dystrophy

Drug development within the rare disease space can be tricky. In many cases, there is a general lack of research and understanding around these conditions; as a result, many…

Source: https://pixabay.com/en/stairway-staircase-stairs-outdoors-828883/

MDA Clinical & Scientific Conference: Givinostat Improves Physical Function and Mobility in DMD

Post author:Jessica Lynn
Post published:April 3, 2023
Post category:Duchenne Muscular Dystrophy

The Muscular Dystrophy Association (MDA) recently held its MDA Clinical & Scientific Conference in March 2023. During the conference, stakeholders in the community discussed research trends and clinical practices associated…

rawpixel / Pixabay

4-Year Proof-of-Concept Data Validates SRP-9001 for DMD

Post author:Jessica Lynn
Post published:March 31, 2023
Post category:Duchenne Muscular Dystrophy

The Muscular Dystrophy Association (MDA) held its MDA Clinical & Scientific Conference from March 19-22, 2023 to share research, cutting-edge medical advancements, and clinical care practices within the muscular…

source: shutterstock.com

Delhi High Court Calls for Duchenne Muscular Dystrophy Clinical Trial Funding

Post author:James Moore
Post published:March 30, 2023
Post category:Duchenne Muscular Dystrophy

According to a story from MENAFN, the Delhi High Court recently issued a directive to the National Consortium for Research, Development, and Therapeutics for Rare Diseases to convene and move…

source: shutterstock.com

“Napa in Miami” Wine Tasting and Auction Raised $1.2M+ for DMD Research

Post author:Jessica Lynn
Post published:March 28, 2023
Post category:Duchenne Muscular Dystrophy

CureDuchenne, the nation’s leading nonprofit dedicated to funding a cure for Duchenne muscular dystrophy (DMD) and a Patient Worthy partner, held its first “Napa in Newport” event in 2015;…

source: pixabay.com

How PepGen’s Jane Larkindale and Alayna Tress Advocate for Patient-Centricity in Rare Disease Drug Development

Post author:Patient Worthy Contributor
Post published:March 1, 2023
Post category:Duchenne Muscular Dystrophy Myotonic Dystrophy

Contributed by Jane Larkindale and Alayna Tress While millions of people globally are living with a rare disease, patients often find it difficult to feel seen or heard throughout their…

Alexas_Fotos / Pixabay

In Utero DMD Therapy Was Unsuccessful in Murine Models

Post author:Jessica Lynn
Post published:December 15, 2022
Post category:Duchenne Muscular Dystrophy

The current standards-of-care for those with Duchenne muscular dystrophy (DMD) include corticosteroids, immunosuppressive treatments, physical therapy, and occupational therapy (among others). Gene therapy has the potential to change the treatment…

source: pixabay.com

DYNE-251 Earns Fast Track Designation for DMD

Post author:Jessica Lynn
Post published:November 17, 2022
Post category:Duchenne Muscular Dystrophy

According to an October 31, 2022 press release from muscle disease company Dyne Therapeutics, Inc. ("Dyne"), the company's therapeutic candidate DYNE-251 earned Fast Track designation from the FDA for Duchenne…

Photo courtesy of CureDuchenne

CureDuchenne Has Launched a Certified Occupational Therapist Program: An Interview with Jennifer Wallace Valdes, PT (Pt. 2)

Post author:Jessica Lynn
Post published:November 10, 2022
Post category:Duchenne Muscular Dystrophy Timely

Before you read on, don't forget to check out Part 1 of our interview with Jennifer Wallace Valdes, PT. In Part 1, Jennifer discusses her background, what Duchenne muscular dystrophy (DMD) is,…

Photo courtesy of CureDuchenne

CureDuchenne Has Launched a Certified Occupational Therapist Program: An Interview with Jennifer Wallace Valdes, PT (Pt. 1)

Post author:Jessica Lynn
Post published:November 9, 2022
Post category:Duchenne Muscular Dystrophy Timely

Occupational therapy for individuals with Duchenne muscular dystrophy (DMD) requires a unique and specialized approach to maximize benefit and reduce harm. It is incredibly important that those performing occupational therapy…

Rare Community Profiles: Destroy Duchenne: How Elijah Stacy is Working to Improve the Lives of People with Duchenne Muscular Dystrophy

Death of Muscular Dystrophy Patient Potentially Caused by Adeno-Associated Virus That Delivered Gene Therapy

CureDuchenne Launches the CureDuchenne Caregiver Course to Support Caregivers of People with DMD

NS-089/NCNP-02 Nabs Orphan Drug Designation for DMD

SAT-3153 Earns Orphan Drug Designation for DMD

These Clinical Trials Could be Game Changers in the Latter Half of 2023

A Top FDA Official Overrules Reviewers’ Rejection of Sarepta’s DMD Gene Therapy

Rare Community Profiles: A Partnership Between CureDuchenne and PicnicHealth Bolsters the Power of CureDuchenne Link

Duchenne Muscular Dystrophy Patient Dies in Gene Therapy Trial

MP1032 Earns Orphan Drug Designation for DMD

Sarepta’s Duchenne Gene Therapy Drug Cleared for Accelerated Approval

Rare Community Profiles: CureDuchenne Clinic in Greater Dallas Area Aims to Improve Treatment Accessibility and Care for Underserved Families with DMD and BMD

Teen with DMD Finishes 12th Flying Pig Marathon

Experimental Duchenne Muscular Dystrophy Treatment Earns Fast Track Designation

MDA Announces Research Grants for Duchenne Muscular Dystrophy

FDA Grants Orphan Drug Designation to DYNE-251 for DMD

MDA Clinical & Scientific Conference: Givinostat Improves Physical Function and Mobility in DMD

4-Year Proof-of-Concept Data Validates SRP-9001 for DMD

Delhi High Court Calls for Duchenne Muscular Dystrophy Clinical Trial Funding

“Napa in Miami” Wine Tasting and Auction Raised $1.2M+ for DMD Research

How PepGen’s Jane Larkindale and Alayna Tress Advocate for Patient-Centricity in Rare Disease Drug Development

In Utero DMD Therapy Was Unsuccessful in Murine Models

DYNE-251 Earns Fast Track Designation for DMD

CureDuchenne Has Launched a Certified Occupational Therapist Program: An Interview with Jennifer Wallace Valdes, PT (Pt. 2)

CureDuchenne Has Launched a Certified Occupational Therapist Program: An Interview with Jennifer Wallace Valdes, PT (Pt. 1)

Let’s Work Together!

Keep Up to Date

Learn More