Updates

May 6, 2021May 6, 2021
CIFFREO Trial of Potential DMD Gene Therapy Delayed in US

Pfizer has announced a delay in opening U.S. sites for its Phase 3 clinical trial CIFFREO, evaluating the investigational gene therapy PF-06939926 in boys ... Read more
May 4, 2021May 4, 2021
Positive Results Reported From Phase 2 Study of SRP-5051 in DMD

Sarepta Therapeutics has announced positive results from a global study evaluating its investigational treatment SRP-5051 in people with Duchenne muscular dystrophy (DMD) who are amenable ... Read more
April 29, 2021April 29, 2021
Couple with FSHD Relish Shared Independence, Advocacy Work

Ray Jordan and Cyndi Segroves, a married couple with facioscapulohumeral muscular dystrophy (FSHD), haven’t let their disability get in the way of anything. They’ve traveled ... Read more
April 28, 2021April 26, 2021
After Falling, I Pick Myself Up and Bend My Armor Back Into Shape

The glint of armor demands attention. The jousting lance was slung to the side of a great black horse. The rider, shield in hand, mounted ... Read more
April 27, 2021April 27, 2021
New Tool Shows Profound Metabolic Changes in DMD Patients’ Muscles

The muscles of people with Duchenne muscular dystrophy (DMD) show profound impairment, with changes in fatty molecules and energy metabolism, as compared with those of ... 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

CIFFREO Trial of Potential DMD Gene Therapy Delayed in US

Positive Results Reported From Phase 2 Study of SRP-5051 in DMD

Couple with FSHD Relish Shared Independence, Advocacy Work

After Falling, I Pick Myself Up and Bend My Armor Back Into Shape

New Tool Shows Profound Metabolic Changes in DMD Patients’ Muscles

Why It’s So Important to Find Your People

#AANAM – Presenter Compares Features of DM1 and DM2

#AANAM – Pregnancy May Worsen Muscle Weakness in LGMDR9 Patients

Quick Approval for COVID Vaccines a Concern, Rare Disease Patients Say

#AANAM – Corticosteroids Aid Muscle Mass in BMD and LGMD, Trials Needed

#AANAM – Investigational DM1 Therapy Shows Promise

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How I’ve Managed Constipation as Someone With Duchenne

EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US

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

#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

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

CIFFREO Trial of Potential DMD Gene Therapy Delayed in US

Positive Results Reported From Phase 2 Study of SRP-5051 in DMD

Couple with FSHD Relish Shared Independence, Advocacy Work

After Falling, I Pick Myself Up and Bend My Armor Back Into Shape

New Tool Shows Profound Metabolic Changes in DMD Patients’ Muscles

Why It’s So Important to Find Your People

#AANAM – Presenter Compares Features of DM1 and DM2

#AANAM – Pregnancy May Worsen Muscle Weakness in LGMDR9 Patients

Quick Approval for COVID Vaccines a Concern, Rare Disease Patients Say

#AANAM – Corticosteroids Aid Muscle Mass in BMD and LGMD, Trials Needed

#AANAM – Investigational DM1 Therapy Shows Promise

Subscribe to our mailing list

How I’ve Managed Constipation as Someone With Duchenne

EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US

DuchenneXchange Launches Mobile, Desktop App for DMD Community

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

#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

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