Updates

February 17, 2020February 13, 2020
As Adults with DMD, We Have a Responsibility to Mentor Younger Generations

Adults with Duchenne muscular dystrophy (DMD) should help younger generations through the difficult parts of growing up with a rare muscular disease. They need ... Read more
February 17, 2020February 17, 2020
Registration for PPMD Advocacy Conference Now Open

Registration for the upcoming Parent Project Muscular Dystrophy (PPMD) Advocacy Conference is now open. During the conference, which will be held March 8–10 in Washington, ... Read more
February 14, 2020February 14, 2020
TAS‐205 Safe, May Slow Muscle Loss in DMD Patients, Phase 2 Data Suggest

Oral treatment with TAS-205 is safe, and may delay muscle deterioration in patients with Duchenne muscular dystrophy (DMD), an early Phase 2 clinical trial ... Read more
February 13, 2020February 12, 2020
Addressing the Funding Crisis for Social Security

For those of you who follow my column, I have some good news: Sammy Eikahldit is back. My alter ego, Sammy, first appeared in ... Read more
February 13, 2020February 13, 2020
CureDuchenne Teams Up with Vivli to Share DMD Clinical Trial Data

Seeking to improve treatment of Duchenne muscular dystrophy (DMD), CureDuchenne has teamed up with Vivli to make clinical trial data available to researchers around the ... Read more

News

FDA Accepts New Drug Application for Potential DMD Treatment Viltolarsen
February 12, 2020February 12, 2020
The U.S. Food and Drug Administration (FDA) accepted NS Pharma‘s new drug application for viltolarsen, ...
News

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
February 11, 2020February 11, 2020
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 ...
News

BIND Project Focuses on Brain Effects of Muscular Dystrophies
February 11, 2020February 11, 2020
A new project called BIND — Brain Involvement  iN  Dystrophinopathies — seeks to better characterize ...
News

World Duchenne Organization Takes Lead in European Efforts to Better Treat DMD
February 10, 2020February 12, 2020
Eighty delegates from 37 countries recently met in Athens for the 2019 Duchenne Patient ...
News

Orsini Specialty Pharmacy Now Provides Vyondys 53 to Treat Duchenne
February 7, 2020February 7, 2020
Orsini Pharmaceutical Services is now providing Sarepta Therapeutics’ newly approved therapy Vyondys 53 (golodirsen) ...
News

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
February 6, 2020February 6, 2020
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan ...

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From Care Centers to Research Awards and Summer Camp, MDA Leads by Helping Others
February 6, 2020February 6, 2020
Expecting to award roughly $18 million in grants in 2020 to support research across ...
Combo Treatments, Gene Therapies and What’s Ahead for Muscular Dystrophy: Talk with MDA
February 6, 2020February 6, 2020
Gene therapies — after a tragedy kicked them back to the lab some two ...
Blocking IGF2R Protein Could Be Therapeutic Strategy, Improve Muscle Strength in DMD, Mouse Study Suggests
February 5, 2020February 5, 2020
Difficulties Strengthen Interabled Relationships
February 4, 2020February 3, 2020
Gene Editing Restores Dystrophin in Pig Model of Duchenne and Patient Cells, Study Reports
February 4, 2020February 4, 2020
Developing My Talents Has Helped Me to Thrive
February 3, 2020January 30, 2020

CITGO Marketer CN Brown Raises More Than $300K for MDA in 2019
February 3, 2020February 3, 2020
Longtime Muscular Dystrophy Association (MDA) supporter CN Brown Company, a CITGO Petroleum Corporation (CITGO) ...
FDA Grants Orphan Drug Status to Losmapimod as Potential FSHD Therapy
January 31, 2020January 31, 2020
Nonprofits RARE Courage, All Wheels Up Partner on Survey Seeking to Improve Air Travel for Wheelchair Users
January 30, 2020February 7, 2020
Now Is the Best Time to Set 2020 Goals
January 30, 2020January 28, 2020

Cx43 Protein May Be Key to Preventing Heart Disease in DMD, Mouse Study Suggests
January 30, 2020January 30, 2020
Altering connexin 43 (Cx43) — a protein involved in heart function — in cardiac ...
Saphyr System to Aid GeneDx in Detecting Mutations Causing Various Muscular Dystrophies
January 29, 2020January 29, 2020
New York Pilot Program Uses PerkinElmer’s Recently Approved DMD Screening Test for Newborns
January 27, 2020January 27, 2020

News

Genethon and Sarepta Extend Collaboration to Develop DMD Gene Therapy
January 24, 2020January 24, 2020
Genethon and Sarepta Therapeutics have extended their original agreement to develop GNT0004, a potential gene ...
News

Enrollment Starts for Phase 2/3 Trial of Oral Therapy for Congenital DM1
January 22, 2020January 22, 2020
AMO Pharma has begun enrollment for a Phase 2/3 trial that will assess the ...
columns, You, Me and MD - a Column by Leah Leilani

Joining a Support Group Can Make a Difference
January 21, 2020January 17, 2020
As a kid, I found it difficult to bond with other children with muscular ...
columns, Hawk's-Eye View - a Column by Hawken Miller

A Positive Attitude Makes a World of Difference When Coping with Duchenne
January 20, 2020January 16, 2020
The other day, I was sitting in Haute Cakes Caffe chowing down on a ...
News

Cure Rare Disease’s Rich Horgan Pioneers Customized Medicine to Treat Brother with DMD
January 20, 2020January 20, 2020
Rich Horgan’s younger brother, Terry Horgan, is the inspiration behind Cure Rare Disease, a ...
News

Duchenne UK Partners with Catabasis to Support Phase 2 Trial of Edasalonexent in DMD
January 17, 2020January 17, 2020
Duchenne UK is partnering with Catabasis Pharmaceuticals to provide financial support for MoveDMD, a Phase 2 ...
columns, Living, Learning, Thriving - a Column by Ralph Yaniz

Society Is Missing the True Definition of Inclusion
January 16, 2020January 15, 2020
My experiences writing this column over the past year have been fabulous. But it ...
News

Montana Rancher’s ‘Calves to Cure DMD’ Funds Duchenne Research
January 15, 2020January 15, 2020
Montana, the fourth-largest U.S. state in area, also is one of the least-populated — ...
columns, You, Me and MD - a Column by Leah Leilani

Regaining Control of My Life with The Happy Planner
January 14, 2020January 10, 2020
Most people with a chronic illness inevitably learn to expect the unexpected with their ...

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

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