muscular dystrophy – Page 3

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…

source: pixabay.com

EDG-5506 Shows Benefits for Becker Muscular Dystrophy, Study Data Shows

Post author:Jessica Lynn
Post published:October 21, 2022
Post category:Becker muscular dystrophy

According to an article from Marisa Wexler in Muscular Dystrophy News, promising study data is available from the Phase 1 ARCH study. Within the study, researchers evaluated EDG-5506, an…

source: pixabay.com

Research Suggests that DMD Begins in Myoblasts

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

In the past, myofibers (terminally differentiated post-mitotic cells that help make up muscle tissue) have been implicated in the development and progression of Duchenne muscular dystrophy (DMD). Basically, researchers have…

Editor’s Choice: A New CRISPR Trial, Revelations About Zika-Related GBS, and Parents Taking Action

Post author:James Moore
Post published:September 8, 2022
Post category:Duchenne Muscular Dystrophy Guillain Barré syndrome Guillain Barre Syndrome Zika Virus

Happy Thursday! This week, we have details on the beginnings of a new trial testing CRISPR gene editing for Duchenne muscular dystrophy, how parents can play a critical role in…

September 7 is World Duchenne Awareness Day: Levi’s Story

Post author:James Moore
Post published:September 7, 2022
Post category:Duchenne Muscular Dystrophy

September 7 is World Duchenne Day, a day to help spread awareness about Duchenne muscular dystrophy among the general public and in the medical field. In recognition of this day,…

igorovsyannykov / Pixabay

“Good Bad Things” Movie Features Man with FSHD

Post author:Jessica Lynn
Post published:September 1, 2022
Post category:Facioscapulohumeral muscular dystrophy 1a

Actor Max Adler is known for a variety of roles in films and TV shows such as Glee, Saugatuck Cures, and Switched at Birth. But did you know that…

Source: pixabay.com

How Cubs Player Frank Schwindel Supports a Boy with a Rare and Unnamed Condition

Post author:Jessica Lynn
Post published:July 29, 2022
Post category:Rare Disease

When Ryker Colón was first born, his parents Eddie and Brandi noticed something potentially concerning. Their young son had a chronic cough - and nobody could seem to figure out…

F1Digitals / Pixabay

First Patient Enrolled in Losmapimod Trial for FSHD

Post author:Jessica Lynn
Post published:July 8, 2022
Post category:Facioscapulohumeral muscular dystrophy 1a

In a news release, biopharmaceutical company Fulcrum Therapeutics, Inc. ("Fulcrum") shared that the first patient was dosed in the Phase 3 REACH study. Within the study, researchers are evaluating…

source: pixabay.com

Boy with DMD Finishes his 11th Flying Pig Marathon

Post author:Jessica Lynn
Post published:May 12, 2022
Post category:Duchenne Muscular Dystrophy

On the first Sunday in May, Cincinnati, OH hosts the Flying Pig Marathon – its 26.2 mile race. This year marked the 24th annual event. But for 17-year-old Diego Ramirez,…

mohamed_hassan / Pixabay

New Research on Gene Therapies for Limb-Girdle Muscular Dystrophy 2B

Post author:Alyssa Stevens
Post published:February 17, 2022
Post category:Limb-girdle muscular dystrophy Limb-girdle muscular dystrophy- type 2B Muscular Dystrophy

According to a recent article, Dr. Jyoti K. Jaiswal shared his research pertaining to gene therapies to be used to treat limb-girdle muscular dystrophy 2B (LGMD2B). Limb-Girdle Muscular Dystrophy (LGMD)…

[Source: pixabay.com]

Study Finds Blocking Sphingolipids Counteracts Muscular Dystrophy in Mice Models

Post author:Alyssa Stevens
Post published:February 9, 2022
Post category:Duchenne Muscular Dystrophy Muscular Dystrophy

According to a recent article, a research team discovered that the inhibition of sphingolipid synthesis on mice models of Duchenne muscular dystrophy can counteract the certain symptoms of the disease.…

Patient Story: Parental Support on DMD Journey

Post author:Kendall Mason
Post published:January 31, 2022
Post category:Duchenne Muscular Dystrophy Muscular Dystrophy

Our parents can be some of our strongest supporters, and Hawken Miller knows this firsthand. He recently published an article in Muscular Dystrophy News Today detailing the love and support…

source: pixabay.com

The House Passes a New Act Which Will Accelerate Research for ALS and Related Diseases

Post author:Trudy Horsting
Post published:January 14, 2022
Post category:Amyotrophic Lateral Sclerosis Muscular Dystrophy

Three organizations focused on amytrophic lateral sclerosis (ALS) as well as other rare neurodegenerative diseases, have just celebrated the House of Representatives' passage of the ACT for ALS (H.R.3537/S.1813). The…

source: pixabay.com

RGX-202 for DMD Earns Orphan Drug Designation

Post author:Jessica Lynn
Post published:December 1, 2021
Post category:Duchenne Muscular Dystrophy

According to a November 23 article from Muscular Dystrophy News, the FDA recently granted Orphan Drug designation to clinical-stage biotechnology company Regenxbio's experimental gene therapy candidate, RGX-202. The gene therapy,…

fernandozhiminaicela / Pixabay

AOC 1001 for DM1 Earns Fast Track Designation

Post author:Jessica Lynn
Post published:November 18, 2021
Post category:Myotonic Dystrophy

Currently, there are no therapeutic options available for patients with myotonic dystrophy type 1 (DM1). However, that may soon change. According to Marketwatch, the FDA recently granted Fast Track…

https://unsplash.com/photos/lCPhGxs7pww

Clinical Trial of Vamorolone Receives Orphan Grant Funding

Post author:Kendall Mason
Post published:October 5, 2021
Post category:Becker muscular dystrophy Duchenne Muscular Dystrophy Muscular Dystrophy

Funding for clinical trials can be an issue, especially for rare disease studies. Luckily, there are programs that exist to help with the financials, such as the "Clinical Studies of…

source: pixabay.com

ICYMI: Givinostat Stops BMD Muscle Shrinkage, Study Shows

Post author:Jessica Lynn
Post published:August 9, 2021
Post category:Becker muscular dystrophy Muscular Dystrophy

For years, researchers have evaluated Italfarmaco's givinostat for patients with Duchenne muscular dystrophy (DMD). In fact, a Phase 2/3 clinical trial showed that long-term givinostat treatment helped slow respiratory decline…

Study Shows How to Better Predict Pacemaker Needs in Myotonic Dystrophy Patients

Post author:Trudy Horsting
Post published:August 2, 2021
Post category:Charcot-Marie-Tooth disease Myotonic Dystrophy

A recent study has examined what risk factors exist for patients diagnosed with myotonic dystrophy, the most common of all muscular dystrophies. This is a progressive, hereditary condition. Regarding cardiac…

source: pixabay.com

Webinar: Using the Rare Disease Cures Accelerator for a DMD Progression Model

Post author:James Moore
Post published:July 28, 2021
Post category:Duchenne Muscular Dystrophy Rare Disease

On July 21, 2021, Patient Worthy attended an online webinar presentation titled "How RDCA-DAP Can Help Inform Optimal Trial Design in Progressive Rare Disease." Organized by the Critical Path Institute…

PublicDomainPictures / Pixabay

Gene Editing and the Future of FSHD

Post author:Jessica Lynn
Post published:July 26, 2021
Post category:Facioscapulohumeral muscular dystrophy 1a

Genetic editing has the potential to address and treat a variety of genetic conditions. Recently, researchers explored gene editing as a potential therapeutic option for patients with facioscapulohumeral muscular dystrophy…

Source: Pixabay.com

Phase 2 Trial Begins to Evaluate Pitolisant for DM1

Post author:Jessica Lynn
Post published:July 26, 2021
Post category:Myotonic Dystrophy

At the end of June 2021, pharmaceutical company Harmony Biosciences Holdings, Inc. ("Harmony") shared the initiation of a Phase 2 clinical trial via news release . During the trial, the…

source: pixabay.com

Cholesterol Metabolism Could Be DMD Therapeutic Target

Post author:Jessica Lynn
Post published:July 2, 2021
Post category:Duchenne Muscular Dystrophy

According to Muscular Dystrophy News, new research suggests that the cholesterol metabolic pathway could offer a potential therapeutic target for patients with Duchenne muscular dystrophy (DMD). Following microRNA analysis, researchers…

source: pixabay.com

Modified Base Editing Could Correct DMD Mutations

Post author:Jessica Lynn
Post published:June 30, 2021
Post category:Duchenne Muscular Dystrophy

Genome editing is a topic which has been on the forefront of medical discussion for years. After all, gene editing allows for the potential to treat - or even cure…

Cure Rare Disease is Helping Families Impacted by DMD

Post author:Kendall Mason
Post published:June 3, 2021
Post category:Duchenne Muscular Dystrophy

Cure Rare Disease is a nonprofit organization that aims to help those who are impacted by rare diseases. Currently, the Boston-based company is planning a fundraiser to celebrate all of…

PhotoLizM / Pixabay

FDA Clears LION-101 IND for LGMD21/R9

Post author:Jessica Lynn
Post published:June 1, 2021
Post category:Limb-girdle muscular dystrophy

In a press release from late May 2021, AAV gene therapy company and Bayer AG subsidiary Asklepios BioPharmaceutical, Inc. ("AskBio") shared that the FDA approved its Investigational New Drug (IND)…

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

EDG-5506 Shows Benefits for Becker Muscular Dystrophy, Study Data Shows

Research Suggests that DMD Begins in Myoblasts

Editor’s Choice: A New CRISPR Trial, Revelations About Zika-Related GBS, and Parents Taking Action

September 7 is World Duchenne Awareness Day: Levi’s Story

“Good Bad Things” Movie Features Man with FSHD

How Cubs Player Frank Schwindel Supports a Boy with a Rare and Unnamed Condition

First Patient Enrolled in Losmapimod Trial for FSHD

Boy with DMD Finishes his 11th Flying Pig Marathon

New Research on Gene Therapies for Limb-Girdle Muscular Dystrophy 2B

Study Finds Blocking Sphingolipids Counteracts Muscular Dystrophy in Mice Models

Patient Story: Parental Support on DMD Journey

The House Passes a New Act Which Will Accelerate Research for ALS and Related Diseases

RGX-202 for DMD Earns Orphan Drug Designation

AOC 1001 for DM1 Earns Fast Track Designation

Clinical Trial of Vamorolone Receives Orphan Grant Funding

ICYMI: Givinostat Stops BMD Muscle Shrinkage, Study Shows

Study Shows How to Better Predict Pacemaker Needs in Myotonic Dystrophy Patients

Webinar: Using the Rare Disease Cures Accelerator for a DMD Progression Model

Gene Editing and the Future of FSHD

Phase 2 Trial Begins to Evaluate Pitolisant for DM1

Cholesterol Metabolism Could Be DMD Therapeutic Target

Modified Base Editing Could Correct DMD Mutations

Cure Rare Disease is Helping Families Impacted by DMD

FDA Clears LION-101 IND for LGMD21/R9

Let’s Work Together!

Keep Up to Date

Learn More