Updates

July 10, 2020July 10, 2020
National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, ... Read more
July 9, 2020July 9, 2020
UK Renews Puldysa Early Access Program for Duchenne Patients

Oral Puldysa (idebenone), Santhera Pharmaceuticals‘ investigational treatment for breathing problems caused by Duchenne muscular dystrophy (DMD), will be available to eligible patients in ... Read more
July 7, 2020July 7, 2020
FDA Asked to Approve Casimersen, Exon 45 Skipping DMD Therapy

Sarepta Therapeutics has submitted a full application to the U.S. Food and Drug Administration (FDA) seeking approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) ... Read more
July 6, 2020July 3, 2020
Don’t Feel Bad About Calling Me ‘Inspiring’

In the disabled community, many people discuss the negative implications of being called “inspiring.” Some have said it’s objectifying, reduces our humanness, or congratulates ... Read more
July 2, 2020July 7, 2020
Coronavirus Relief Fund Created to Help DMD Patients, Families

The COVID-19 pandemic has forced many to shelter at home, caused millions of layoffs, and tightened budgets. But for families affected by Duchenne muscular ... Read more

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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

UK Renews Puldysa Early Access Program for Duchenne Patients

FDA Asked to Approve Casimersen, Exon 45 Skipping DMD Therapy

Don’t Feel Bad About Calling Me ‘Inspiring’

Coronavirus Relief Fund Created to Help DMD Patients, Families

CHMP Recommends Translarna Label Update That Enables Treating DMD Patients Unable to Walk

Looking Backward but Living Life Forward With MD

Phase 1 Trial of Potential Oral Treatment for LGMD2i Doses 1st Volunteer

Collaboration Provides Cough Assist Machines to British Columbia Residents With Neuromuscular Disease

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

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Creativity Allows Me to Surpass Any Physical Limits

Sarepta’s Gene Therapy for DMD Showing to Safety, Efficacy at One Year

MDA Holds Q&A on Navigating COVID-19, and Area Reopenings, Safely

Duchenne Has Forced Me to Reflect on My Own Mortality

Viltolarsen Earns European Orphan Drug Designation as DMD Treatment

Coalition Will Address Racial Disparities in Rare Disease Communities

SRP-9003 Gene Therapy for LGMD2E Shows Benefits After 1 Year

Learning to Accept My Mental Illness Through Creativity

Santhera and Rutgers Partner on Gene Therapy Work for LAMA2 MD

$1M Gene Therapy Research Grant Launched by PPMD, Duchenne UK

What I’ve Learned from Starting a Twitch Channel

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Let’s Aim to Achieve True Global Accessibility

Viltolarsen Raises Dystrophin Levels, Aids Muscles in DMD Boys in Phase 2 Trial

Trial Supports ATL1102’s Safety in Improving Muscle Strength, Function in DMD Boys

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

SIDEROS Phase 3 Trial of Puldysa in Duchenne Patients Completes Enrollment

Pfizer’s DMD Gene Therapy Continues to Show Promise, Phase 1b Trial Shows

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

CAP-1002 Improves Muscle, Lung and Heart Function in Young DMD Patients, HOPE-2 Trial Data Show

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

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National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

UK Renews Puldysa Early Access Program for Duchenne Patients

FDA Asked to Approve Casimersen, Exon 45 Skipping DMD Therapy

Don’t Feel Bad About Calling Me ‘Inspiring’

Coronavirus Relief Fund Created to Help DMD Patients, Families

CHMP Recommends Translarna Label Update That Enables Treating DMD Patients Unable to Walk

Looking Backward but Living Life Forward With MD

Phase 1 Trial of Potential Oral Treatment for LGMD2i Doses 1st Volunteer

Dating in the Era of Coronavirus

Collaboration Provides Cough Assist Machines to British Columbia Residents With Neuromuscular Disease

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

Subscribe to our mailing list

Creativity Allows Me to Surpass Any Physical Limits

Sarepta’s Gene Therapy for DMD Showing to Safety, Efficacy at One Year

MDA Holds Q&A on Navigating COVID-19, and Area Reopenings, Safely

Duchenne Has Forced Me to Reflect on My Own Mortality

Viltolarsen Earns European Orphan Drug Designation as DMD Treatment

Medicare’s Role for People with Disabilities

Coalition Will Address Racial Disparities in Rare Disease Communities

SRP-9003 Gene Therapy for LGMD2E Shows Benefits After 1 Year

Learning to Accept My Mental Illness Through Creativity

Santhera and Rutgers Partner on Gene Therapy Work for LAMA2 MD

$1M Gene Therapy Research Grant Launched by PPMD, Duchenne UK

What I’ve Learned from Starting a Twitch Channel

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Let’s Aim to Achieve True Global Accessibility

Viltolarsen Raises Dystrophin Levels, Aids Muscles in DMD Boys in Phase 2 Trial

Trial Supports ATL1102’s Safety in Improving Muscle Strength, Function in DMD Boys

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

SIDEROS Phase 3 Trial of Puldysa in Duchenne Patients Completes Enrollment

Pfizer’s DMD Gene Therapy Continues to Show Promise, Phase 1b Trial Shows

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

CAP-1002 Improves Muscle, Lung and Heart Function in Young DMD Patients, HOPE-2 Trial Data Show

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

Pin It on Pinterest