Updates

November 11, 2019November 11, 2019
Clinical Trial Program Testing RG6206 in DMD Boys Has Been Discontinued

The clinical program evaluating the safety, tolerability, and efficacy of RG6206 in boys with Duchenne muscular dystrophy (DMD) has been discontinued, according to a letter ... Read more
November 11, 2019November 8, 2019
Attack of the Bloodthirsty Mosquitoes

Fall and winter have always been my favorite seasons. I eagerly await the cooler weather by prematurely washing my sweaters and drinking warm beverages. ... Read more
November 9, 2019November 9, 2019
Local November 9th MDA Disease Education Events Span East, West Coast

The Muscular Dystrophy Association’s (MDA) efforts to support and advocate for a wide range of neuromuscular diseases are felt by patient communities nationwide. While ... Read more
November 8, 2019November 8, 2019
Experimental Cell Therapy Improves Cardiac Function in Mouse Model of DMD, Study Says

Treatment with a cell therapy candidate called dystrophin expressing chimeras (DECs) increased dystrophin levels in heart muscle and improved cardiac function in mice with ... Read more
November 6, 2019November 6, 2019
Investigational Therapy Losmapimod Targets Root Cause of FSHD, Early Study Suggests

The investigational oral treatment losmapimod is able to suppress the toxic activity of the DUX4 gene, the root cause of muscle degeneration in ... Read more

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columns, Hawk's-Eye View - a Column by Hawken Miller

Goals Can Simplify Our Complicated Lives
November 4, 2019October 31, 2019
It can sometimes feel like everything is against us when doctor appointments and physical ...
News

BIIB100 Improves Motor Function, Slows DMD Progression in Animal Models, Study Finds
November 4, 2019November 4, 2019
An experimental therapy called BIIB100 improved motor function and slowed the progression of Duchenne ...
News

PPMD Grants Funding for Research on Cardiac Health in People with Duchenne
November 1, 2019November 1, 2019
Parent Project Muscular Dystrophy (PPMD) has awarded a total of $148,000 to researchers at ...
columns, Living, Learning, Thriving - a Column by Ralph Yaniz

Prioritizing Goals in Life with MD
October 31, 2019October 29, 2019
I have spent my entire career working and volunteering in the nonprofit arena. My ...
News

Translarna Preserves Lung Function, Muscle Strength in DMD Boys, Real-World Data Show
October 30, 2019October 30, 2019
Treatment with Translarna (ataluren) preserves lung function and muscle strength in boys with Duchenne ...
columns, You, Me and MD - a Column by Leah Leilani

I Won’t Give Up My Positive Attitude for Others
October 29, 2019October 25, 2019
Sometimes life with a chronic invisible illness can feel backward and upside down. As ...

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Vamorolone Designated Promising Innovative Medicine in UK for DMD Treatment
October 28, 2019October 28, 2019
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted promising innovative medicine (PIM) ...
Novel Gene Therapy Safely Prevents Muscle Deterioration in Animal Models of DMD, Study Reports
October 25, 2019October 25, 2019
A novel one-time gene therapy that leads to the production of a dystrophin-related protein ...
Vamorolone Extension Trial Supports Muscle Gains in DMD with Fewer Side Effects, Topline Data Show
October 23, 2019October 23, 2019
Adjusting to Life Without My Gallbladder
October 22, 2019October 18, 2019
Friendships Matter: You Don’t Have to Deal with Duchenne Alone
October 21, 2019October 18, 2019
CureDuchenne Developing a Biobank for DMD Research
October 21, 2019October 21, 2019

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0 out of 5 stars. 0 votes.

Average Rating

0 out of 5 stars. 0 votes.

Dyne Therapeutics Supporting FSHD Natural History Study Underway in US, Europe
October 10, 2019October 18, 2019
Dyne Therapeutics announced that it has joined in supporting an ongoing global and observational natural ...
Gene Therapy SRP-9003 Showing ‘Very Encouraging’ Results at 9 Months in Limb Girdle MD, Sarepta Reports
October 9, 2019October 9, 2019
The Right Job Can Help You Find Fulfillment and Purpose
October 7, 2019October 3, 2019

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News

Request to Approve Viltolarsen, Exon 53 Skipping Treatment for Duchenne MD, Before FDA
October 7, 2019October 7, 2019
Nippon Shinyaku filed a request for the approval of viltolarsen (NS-065/NCNP-01) to treat Duchenne muscular dystrophy ...
News

First Baby Tested for Duchenne in PPMD’s Newborn Screening Pilot Program in NY
October 4, 2019October 4, 2019
Parent Project Muscular Dystrophy (PPMD) announced the screening of a first baby for Duchenne muscular ...
columns, Living, Learning, Thriving - a Column by Ralph Yaniz

Opportunities Abound for Travel and Accessibility
October 3, 2019October 10, 2019
My first column for Muscular Dystrophy News Today focused on the excitement of starting ...
News

FDA Awards $1M to Phase 2 Study of Ifetroban in Treating Heart Disease Linked to Duchenne MD
October 2, 2019October 2, 2019
Cumberland Pharmaceuticals announced that it has been awarded a $1 million grant by the ...
columns, You, Me and MD - a Column by Leah Leilani

Honoring Serena Lawrence: How Her Life Changed Mine
October 1, 2019September 27, 2019
In the summer of 2017, I received an email from a woman named Serena ...
30 Days of MD

Day 30 of 30 Days of MD: Introducing Columnist Hawken Miller
September 30, 2019
View this post on Instagram Day 30 of #30DaysOfMD Introducing columnists Hawken Miller! Check ...
News

CureDuchenne Plans Oct. 11-13 ‘Futures’ Meet at California’s Disneyland
September 30, 2019September 30, 2019
About 700 people have registered to attend CureDuchenne’s Futures conference taking place Oct. 11-13 ...
30 Days of MD

Day 29 of 30 Days of MD: Being a mother of twins and MD
September 29, 2019
View this post on Instagram Day 29 of #30daysofMD Introducing Lauren! To begin to ...
30 Days of MD

Day 28 of 30 Days of MD: Rehabilitation Physician
September 28, 2019
View this post on Instagram Day 28 of #30daysofMD Vovanti Jones – Rehabilitation Physician ...

May 21, 2018June 3, 2019

FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E

News

The U.S. Food and Drug Administration recently granted rare pediatric disease designation to Myonexus Therapeutics’ experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or LGMD2E. The announcement was made by Myonexus and Nationwide ... Read more

Updates

Clinical Trial Program Testing RG6206 in DMD Boys Has Been Discontinued

Attack of the Bloodthirsty Mosquitoes

Local November 9th MDA Disease Education Events Span East, West Coast

Experimental Cell Therapy Improves Cardiac Function in Mouse Model of DMD, Study Says

Investigational Therapy Losmapimod Targets Root Cause of FSHD, Early Study Suggests

Goals Can Simplify Our Complicated Lives

BIIB100 Improves Motor Function, Slows DMD Progression in Animal Models, Study Finds

PPMD Grants Funding for Research on Cardiac Health in People with Duchenne

Prioritizing Goals in Life with MD

Translarna Preserves Lung Function, Muscle Strength in DMD Boys, Real-World Data Show

I Won’t Give Up My Positive Attitude for Others

Subscribe to our mailing list

Vamorolone Designated Promising Innovative Medicine in UK for DMD Treatment

Novel Gene Therapy Safely Prevents Muscle Deterioration in Animal Models of DMD, Study Reports

Vamorolone Extension Trial Supports Muscle Gains in DMD with Fewer Side Effects, Topline Data Show

Adjusting to Life Without My Gallbladder

CureDuchenne Developing a Biobank for DMD Research

Peeling Back the Layers of Societal Barriers for People with Disabilities

Positive Early Results Support Continuation of Phase 2 Trial of ATL1102 for DMD

PPMD Grants to Promote Gene Therapy for Heart Disease and Patient Outcomes Research

Catabasis Joins Jain Foundation to Advance Edasalonexent Development for Dysferlinopathy

Dyne Therapeutics Supporting FSHD Natural History Study Underway in US, Europe

Gene Therapy SRP-9003 Showing ‘Very Encouraging’ Results at 9 Months in Limb Girdle MD, Sarepta Reports

The Right Job Can Help You Find Fulfillment and Purpose

Request to Approve Viltolarsen, Exon 53 Skipping Treatment for Duchenne MD, Before FDA

First Baby Tested for Duchenne in PPMD’s Newborn Screening Pilot Program in NY

Opportunities Abound for Travel and Accessibility

FDA Awards $1M to Phase 2 Study of Ifetroban in Treating Heart Disease Linked to Duchenne MD

Honoring Serena Lawrence: How Her Life Changed Mine

Day 30 of 30 Days of MD: Introducing Columnist Hawken Miller

CureDuchenne Plans Oct. 11-13 ‘Futures’ Meet at California’s Disneyland

Day 29 of 30 Days of MD: Being a mother of twins and MD

Day 28 of 30 Days of MD: Rehabilitation Physician

FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E

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