Updates

March 2, 2021March 2, 2021
Dosing Begins in Phase 2 Trial Testing Oral Therapy BBP-418 for LGMD2i

The first patient has been dosed in a Phase 2 trial testing BBP-418, a potential first oral disease-modifying therapy for limb-girdle muscular dystrophy type 2i ... Read more
February 26, 2021February 26, 2021
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from ... Read more
February 25, 2021February 25, 2021
Exon Skipping Dominates DMD Treatment, But What’s on Horizon?

With the recent approval of Amondys 45 (casimersen) by Sarepta Therapeutics, there are now four exon-skipping therapies available in the U.S. for eligible patients with ... Read more
February 25, 2021February 26, 2021
FDA Approves Amondys 45 for Duchenne Patients With Exon 45 Mutation

The U.S. Food and Drug Administration (FDA) has conditionally approved Amondys 45 — formerly known as casimersen — by Sarepta Therapeutics as the first treatment for people with Duchenne ... Read more
February 23, 2021February 23, 2021
Givinostat Continues to Delay DMD Progression in Long-term Study, New Data Show

Treatment with givinostat alongside corticosteroids continues to delay disease progression — and maintain patients’ ability to walk — in boys with 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 Children’s ... Read more

Updates

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 With Exon 45 Mutation

Givinostat Continues to Delay DMD Progression in Long-term Study, New Data Show

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Yes, We Are Rare, but Please Realize We Are Human First

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

MDA Launches 39th Annual #Shamrocks Fundraiser

How an Albino Direwolf Named Ghost Made Me Think About Being Rare

Deleting Mss51 Gene Reduces Fatigue, Increases Endurance in DMD Mice

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PPMD Receives Award to Improve Neurobehavioral Care in Duchenne

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Translarna Increased Dystrophin Levels in Phase 2 DMD Study

It’s Time for Me to Go Outdoors and Take in the Vitamin D

Isometric Exercise in DMD Found to Improve Boys’ Strength, Function

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

Welcome to the Castle: Facing Down and Defeating Your Fears

New App Plays ‘Pivotal’ Role in Increasing Registry Successes

STRIVE Applications for Duchenne Community Now Open

PPMD Invests $1M in Satellos With Goal of Repairing Muscle Cells in Duchenne

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Why I’m Taking a Break From Playing Video Games

New CureDuchenne Initiative Provides Support, Education on COVID-19 Vaccination

NORD’s Caregiver Respite Program Continues Through Pandemic

A Bittersweet Farewell to My Column Readers

Exon 45 Skipping Therapy for DMD Shows Safety in Small Trial

Dosing Begins in Phase 3 Trial of Pfizer’s Gene Therapy for Duchenne

I Make Sure My Support System Is Strong

DMD Boys Experience Biological, Functional Gains in SRP-9001 Study

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

Why Freelancing Works for My Life With Duchenne

Pivotal Trial Begins for AMO-02 in Steinert Disease

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