muscular dystrophy – Page 2

Knowledge is Power: CureDuchenne Cares Educational Events Aim to Equip Families and Caregivers in the Becker and Duchenne Muscular Dystrophy Communities

Post author:Jessica Lynn
Post published:February 2, 2024
Post category:Duchenne Muscular Dystrophy

CureDuchenne, a global leader in research and patient care for Duchenne muscular dystrophy (DMD), has been working to find and fund a cure for DMD for more than two decades.…

PublicDomainPictures / Pixabay

SGT-003 Granted Orphan Drug Designation for Duchenne Muscular Dystrophy (DMD)

Post author:Jessica Lynn
Post published:January 22, 2024
Post category:Duchenne Muscular Dystrophy

The research landscape for Duchenne muscular dystrophy (DMD) has been steadily increasing. Researchers are exploring more therapies, including gene therapies, that could transform the lives of those with this rare…

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Vamorolone Has Been FDA Approved to Treat Duchenne Muscular Dystrophy

Post author:Rose Duesterwald
Post published:November 27, 2023
Post category:Duchenne Muscular Dystrophy

Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side…

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,…

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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,…

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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…

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FDA Clears Clinical Study to Evaluate BB-301 for Oculopharyngeal Muscular Dystrophy

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

Prior to launching a clinical trial, the FDA must clear an Investigational New Drug (IND) application. This allows the drug to be distributed across state lines, which is important…

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: Maddie Graves Photography

Two Affected with Congenital Muscular Dystrophy Strive for Rare Inclusion

Post author:Patient Worthy Contributor
Post published:July 28, 2023
Post category:Congenital Muscular dystrophy

Congenital muscular dystrophy (CMD) affected individuals, Kelly Berger and Avery Roberts are making their voices heard. Together they hope to tackle misconceptions surrounding disability and give a loud voice…

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…

qimono / Pixabay

ASGCT Annual Meeting: Presentation Shared Data on EPI-321 for FSHD

Post author:Jessica Lynn
Post published:June 12, 2023
Post category:Facioscapulohumeral muscular dystrophy 1a

This year, the American Society of Gene & Cell Therapy (ASGCT) held its 26th annual meeting from May 16 through May 20, 2023. In a news release, biotechnology company…

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…

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…

source: shutterstock.com

Mevalonolactone Treatment Eased Symptoms of Statin Myopathy and a form of LGMD

Post author:Jessica Lynn
Post published:April 7, 2023
Post category:Limb-girdle muscular dystrophy

Statins are a type of drug that can help you to lower your blood cholesterol levels. These drugs work by inhibiting HMG CoA reductase, an enzyme that the body…

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…

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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

“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

AOC 1020 Granted Fast Track Designation for FSHD

Post author:Jessica Lynn
Post published:January 25, 2023
Post category:Facioscapulohumeral muscular dystrophy 1a

A January 2023 news release from biopharmaceutical company Avidity Biosciences, Inc. ("Avidity") shares that the company's therapy, AOC 1020, earned Fast Track designation from the FDA for the treatment of…

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,…

Knowledge is Power: CureDuchenne Cares Educational Events Aim to Equip Families and Caregivers in the Becker and Duchenne Muscular Dystrophy Communities

SGT-003 Granted Orphan Drug Designation for Duchenne Muscular Dystrophy (DMD)

Vamorolone Has Been FDA Approved to Treat Duchenne Muscular Dystrophy

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

FDA Clears Clinical Study to Evaluate BB-301 for Oculopharyngeal Muscular Dystrophy

SAT-3153 Earns Orphan Drug Designation for DMD

Two Affected with Congenital Muscular Dystrophy Strive for Rare Inclusion

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

ASGCT Annual Meeting: Presentation Shared Data on EPI-321 for FSHD

MP1032 Earns Orphan Drug Designation for DMD

Teen with DMD Finishes 12th Flying Pig Marathon

Experimental Duchenne Muscular Dystrophy Treatment Earns Fast Track Designation

Mevalonolactone Treatment Eased Symptoms of Statin Myopathy and a form of LGMD

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

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

AOC 1020 Granted Fast Track Designation for FSHD

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)

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