Updates

August 13, 2020August 13, 2020
FDA Approves Viltolarsen, Now Viltepso, for DMD Patients With Exon 53 Mutation

The U.S. Food and Drug Administration (FDA) has conditionally approved NS Pharma‘s Viltepso (viltolarsen) for people with Duchenne muscular dystrophy (DMD) amenable to exon ... Read more
August 11, 2020August 10, 2020
I’m Sleeping Comfortably Now, Thanks to a New Mattress

Most mitochondrial myopathy patients rely on sleep as therapy for our disease. Yes, rest can be effective at regenerating lost energy, but there’s nothing ... Read more
August 11, 2020August 11, 2020
Patient Feedback Being Sought on Puldysa for Possible NHS Funding

The National Institute for Health and Care Excellence (NICE) is urging patient organizations to gather feedback from those with Duchenne muscular dystrophy (DMD) who ... Read more
August 10, 2020August 10, 2020
New Streaming Channel Showcases Rare Disease Films

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a ... Read more
August 10, 2020August 7, 2020
Losing My Ability to Walk Won’t Stop Me

When I was younger, I couldn’t imagine losing my ability to get up from the ground, run, or play soccer. Now I can’t imagine ... 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

FDA Approves Viltolarsen, Now Viltepso, for DMD Patients With Exon 53 Mutation

I’m Sleeping Comfortably Now, Thanks to a New Mattress

Patient Feedback Being Sought on Puldysa for Possible NHS Funding

New Streaming Channel Showcases Rare Disease Films

Losing My Ability to Walk Won’t Stop Me

MD Advocates Offer Advice for Students With Neuromuscular Disorders Amid COVID-19

FDA Requests More Data, Maintains Clinical Hold on IGNITE DMD Trial of SGT-001 Gene Therapy

The Angels Who’ve Profoundly Affected My Life

A Day on the Hill: Using Our Voices to Lobby Lawmakers

FDA Places SRP-9001, DMD Micro-dystrophin Gene Therapy, on Fast Track

Stem Cell Therapy VTA-110 Named FDA Orphan Drug for DMD Treatment

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Benitec Begins Early Study of BB-301 Gene Therapy for OPMD

Two Imaging Biomarkers Predict Walking Ability in DMD, Useful for Trials

A Good Sense of Humor Makes my Duchenne Diagnosis Easier

NS Pharma Opens Support Hub to Help DMD Patients Access Treatments

Hansa Licenses Imlifidase to Sarepta as Gene Therapy Pre-treatment for DMD, LGMD

Being Independent Within My Capabilities

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

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

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

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FDA Approves Viltolarsen, Now Viltepso, for DMD Patients With Exon 53 Mutation

I’m Sleeping Comfortably Now, Thanks to a New Mattress

Patient Feedback Being Sought on Puldysa for Possible NHS Funding

New Streaming Channel Showcases Rare Disease Films

Losing My Ability to Walk Won’t Stop Me

MD Advocates Offer Advice for Students With Neuromuscular Disorders Amid COVID-19

FDA Requests More Data, Maintains Clinical Hold on IGNITE DMD Trial of SGT-001 Gene Therapy

The Angels Who’ve Profoundly Affected My Life

A Day on the Hill: Using Our Voices to Lobby Lawmakers

FDA Places SRP-9001, DMD Micro-dystrophin Gene Therapy, on Fast Track

Stem Cell Therapy VTA-110 Named FDA Orphan Drug for DMD Treatment

Subscribe to our mailing list

Benitec Begins Early Study of BB-301 Gene Therapy for OPMD

Two Imaging Biomarkers Predict Walking Ability in DMD, Useful for Trials

A Good Sense of Humor Makes my Duchenne Diagnosis Easier

NS Pharma Opens Support Hub to Help DMD Patients Access Treatments

Hansa Licenses Imlifidase to Sarepta as Gene Therapy Pre-treatment for DMD, LGMD

Being Independent Within My Capabilities

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

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

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

Pin It on Pinterest