Updates

April 13, 2021April 13, 2021
FDA Places Pamrevlumab for DMD on Fast Track

The U.S. Food and Drug Administration has granted fast track designation to pamrevlumab, an investigational treatment for Duchenne muscular dystrophy (DMD). The designation is given ... Read more
April 8, 2021April 8, 2021
Keisha Greaves Named MDA’s 2021 National Goodwill Ambassador

The Muscular Dystrophy Association (MDA) has named Keisha Greaves, a fashion entrepreneur and neuromuscular disease advocate from Cambridge, Massachusetts, as its latest National Ambassador. “I have ... Read more
April 6, 2021April 6, 2021
Audentes Becomes Astellas Gene Therapies, Will Advance MD Treatments

Following its acquisition last year by Astellas Pharma, Audentes Therapeutics now has become Astellas Gene Therapies. Named a Gene Therapy Center of Excellence, it will ... Read more
April 5, 2021April 1, 2021
Dungeons and Dragons Helps Me Live Without Duchenne for a Moment

Kriki Cragwalker, a redheaded dwarf from the mountain city of Jagarz Dauk, grasps her enchanted, two-handed greataxe and is circled by a host of ancient ... Read more
April 1, 2021April 1, 2021
Flavocoxid Well-tolerated, Lessens Inflammation in Small DMD Trial

Flavocoxid was well-tolerated and lessened inflammation in a small clinical trial involving boys with Duchenne muscular dystrophy (DMD). Researchers said these results support Flavocoxid’s further ... 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 Children’s Hospital. ... Read more

Updates

FDA Places Pamrevlumab for DMD on Fast Track

Keisha Greaves Named MDA’s 2021 National Goodwill Ambassador

Audentes Becomes Astellas Gene Therapies, Will Advance MD Treatments

Dungeons and Dragons Helps Me Live Without Duchenne for a Moment

Flavocoxid Well-tolerated, Lessens Inflammation in Small DMD Trial

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

What a ‘Typical’ Day Is Like for My Son, Alfie, and Me

Second Phase 3 Study of Pamrevlumab Recruiting Boys With DMD

#MDA2021 – Long-term Vamorolone Slowed Disease Progression in DMD Boys

#MDA2021 – DMD Gene Therapy PF-06939926 Continues to Show Promise

#MDA2021 – Gene Therapy SGT-001 Shows Continued Benefit in DMD Boys

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#MDA2021 – Factors Likely to Predict Wheelchair Use With FSHD1 Detailed

#MDA2021 – LGMD2E Gene Therapy SRP-9003 Shows Benefits After 2 Years

What Having Duchenne Muscular Dystrophy Feels Like

#MDA2021 – Plasma Exchange Enables Effective Gene Therapy Redosing

#MDA2021 — Gene Therapy for DMD Exon Duplication Showing Promise in Trial

#MDA2021 – Patients, Families Discuss Diagnosis, Symptoms of LGMD2i

#MDA2021 – SRP-5051 Shows Promise in Animal Studies, Supporting Phase 2 Trial

Springtime Brings Renewed Opportunity for Activity and Adventure

#MDA2021 — Amondys 45 Safe and Well-tolerated in Phase 1/2 Trial

#MDA2021 — Patients Switched to Emflaza for Efficacy, Tolerability

What I’ve Learned Since My Son’s LAMA2 MD Diagnosis

MDA Engage Offers Free Educational Events

#MDA2021 – MDA Virtual Conference to Feature Latest in Clinical Care, Research

I’m Ready to Go Forward After My 2nd Dose of the COVID-19 Vaccine

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

Vamorolone Trial for DMD Finishes Pivotal First Part, Results Awaited

A Hefty Guitar Impacts My Ability to Keep the Music Going

Dosing Begins in Phase 2 Trial Testing Oral Therapy BBP-418 for LGMD2i

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Exon Skipping Dominates DMD Treatment, But What’s on Horizon?

FDA Approves Amondys 45 for Duchenne Patients Amenable to Exon 45 Skipping

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

FDA Places Pamrevlumab for DMD on Fast Track

Keisha Greaves Named MDA’s 2021 National Goodwill Ambassador

Audentes Becomes Astellas Gene Therapies, Will Advance MD Treatments

Dungeons and Dragons Helps Me Live Without Duchenne for a Moment

Flavocoxid Well-tolerated, Lessens Inflammation in Small DMD Trial

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

What a ‘Typical’ Day Is Like for My Son, Alfie, and Me

Second Phase 3 Study of Pamrevlumab Recruiting Boys With DMD

#MDA2021 – Long-term Vamorolone Slowed Disease Progression in DMD Boys

#MDA2021 – DMD Gene Therapy PF-06939926 Continues to Show Promise

#MDA2021 – Gene Therapy SGT-001 Shows Continued Benefit in DMD Boys

Subscribe to our mailing list

#MDA2021 – Factors Likely to Predict Wheelchair Use With FSHD1 Detailed

#MDA2021 – LGMD2E Gene Therapy SRP-9003 Shows Benefits After 2 Years

What Having Duchenne Muscular Dystrophy Feels Like

#MDA2021 – Plasma Exchange Enables Effective Gene Therapy Redosing

#MDA2021 — Gene Therapy for DMD Exon Duplication Showing Promise in Trial

#MDA2021 – Patients, Families Discuss Diagnosis, Symptoms of LGMD2i

#MDA2021 – SRP-5051 Shows Promise in Animal Studies, Supporting Phase 2 Trial

Springtime Brings Renewed Opportunity for Activity and Adventure

#MDA2021 – Pandemic Added Access Challenges for Patients Surveyed Globally

#MDA2021 — Amondys 45 Safe and Well-tolerated in Phase 1/2 Trial

#MDA2021 — Patients Switched to Emflaza for Efficacy, Tolerability

What I’ve Learned Since My Son’s LAMA2 MD Diagnosis

MDA Engage Offers Free Educational Events

#MDA2021 – MDA Virtual Conference to Feature Latest in Clinical Care, Research

I’m Ready to Go Forward After My 2nd Dose of the COVID-19 Vaccine

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

Vamorolone Trial for DMD Finishes Pivotal First Part, Results Awaited

A Hefty Guitar Impacts My Ability to Keep the Music Going

Dosing Begins in Phase 2 Trial Testing Oral Therapy BBP-418 for LGMD2i

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Exon Skipping Dominates DMD Treatment, But What’s on Horizon?

FDA Approves Amondys 45 for Duchenne Patients Amenable to Exon 45 Skipping

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