Businessman Driven to Cure DMD After a Devastating Family Diagnosis
Ilan Ganot, CEO of Solid Biosciences, cofounded the company with his wife, Annie, after their son, Eytani, was diagnosed with Duchenne in 2012.
DMD Features
Duchenne Gene Therapy Pioneer Dr. Jerry Mendell Reflects on a Lifetime of Research
Jerry R. Mendell, MD, professor of neurology and pediatrics at Nationwide Children’s Hospital has devoted his life’s work to curing DMD.
Poster Child for ‘Right to Try’ Movement Marks 5 Years on Novel DMD Treatment
A video of Jordan McLinn, who has DMD, went viral when he tried to embrace then-President Trump before he signed the “Right to Try” bill at the White House.
Black Father Highlights Challenges Faced by Rare Disease Patients From Minority Groups
Jay Griffin, whose son Franklin has DMD, knows all too well what it’s like to be a member of a minority group and in search of help with a rare disease.
MyoGene Bio Founder Dr. Courtney Young Inspired by Cousin’s Duchenne Diagnosis
Courtney Young, PhD, developed her startup, MyoGene Bio, to find a cure for Duchenne muscular dystrophy, which plagues her young cousin.
Pilot Projects in 3 States Show the Promise of Screening Newborns for Duchenne
Pilot programs in New York, Massachusetts, and North Carolina testing babies for DMD may bolster the case for nationwide screening for this rare disease.
MDA 2022 Conference to Focus on Promising Therapies for SMA, DMD
Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) will take center stage at the first in-person annual gathering of the Muscular Dystrophy Association (MDA) since March 2019.
Families of Patients With DMD Fight for Awareness in Poverty-Stricken Ukraine
Families of boys with Duchenne muscular dystrophy have a hard time accessing lifesaving therapies and services in Ukraine, Europe’s poorest country.
CureDuchenne to Highlight Latest Developments in Gene Therapy, Editing
This year’s CureDuchenne Futures conference features a panel on exon skipping as well as sessions on the family’s role in research, quality of life, cognition in Duchenne, and defining happiness.
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Israel Emerges as Global Powerhouse in Rare Disease Research
When it comes to rare disorders, Israel—a New Jersey-sized nation of 9.3 million that’s made headlines this year for its highly successful coronavirus vaccination campaign—is clearly an emerging powerhouse.
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