April 20, 2018April 20, 2018
#AAN2018 – Clinical Trials Re-Analysis Shows Emflaza Potential to Preserve Motor Function in Boys with DMD
News
Treatment with Emflaza (deflazacort) is better at preserving motor function in patients with Duchenne muscular dystrophy (DMD) ... Read more
April 18, 2018April 18, 2018
#AAN2018 — Exondys 51 Found to Be Safe in Young Infant with DMD, Preliminary Data Shows
News
Treatment with Exondys 51 (eteplirsen) was found to be safe and well-tolerated in a very young infant ... Read more
April 17, 2018April 16, 2018
Noticing the Blessings in Everyday Life
columns, You, Me and MD - a Column by Leah Leilani
Chronic illness can dramatically alter life perspective. If there’s anything I’ve learned from having mitochondrial myopathy, it’s gratitude for ... Read more
April 16, 2018April 16, 2018
UT Researcher, with Cure Duchenne Support, Launches Company to Treat DMD Using CRISPR/Cas9 Technology
News
Eric Olson, a molecular biologist, has spent more than half his life in the lab. Now, the 62-year-old University ... Read more
April 13, 2018April 12, 2018
NIH Agency Pioneers Collaborative Research into Rare Diseases
News
A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed ... Read more
April 13, 2018April 13, 2018
Pfizer Launches Phase 1b Clinical Trial for Mini-Dystrophin Gene Therapy to Treat DMD
News
Pfizer, Inc began a Phase 1b clinical trial (NCT03362502) for its investigational mini-dystrophin gene therapy product, PF-06939926, in patients with ... Read more
April 10, 2018April 9, 2018
Breaking the Stigma of Asking for Help
columns, You, Me and MD - a Column by Leah Leilani
A lot of people think that asking for help is a piece of cake. A walk in the park. ... Read more
April 10, 2018April 11, 2018
Genetic Compensation Mechanism Could Aid in Treatment of Muscle Disorders, Study Suggests
News
A genetic compensation mechanism discovered in zebra fish could have implications in the treatment of muscle diseases, a recent ... Read more
April 8, 2018April 9, 2018
PPMD Names PerkinElmer to Conduct Genetic Tests for Its Decode Duchenne Program
News
Parent Project Muscular Dystrophy (PPMD) has selected PerkinElmer to provide genetic testing for its Decode Duchenne program, which offers testing and ... Read more
April 5, 2018April 6, 2018
Santhera’s Omigapil Found Safe and Well-Tolerated in Young CMD Patients in Phase 1 Trial
News
Omigapil, an investigational therapy by Santhera Pharmaceuticals, proved to be safe and well-tolerated in children and adolescents with congenital muscular ... Read more
April 4, 2018April 4, 2018
Connecticut Neurologist Upbeat on New Therapies for DMD, Other Disorders
News
Gyula Acsadi is an expert in three of the world’s most expensive rare illnesses to treat: Duchenne muscular dystrophy ... Read more
April 4, 2018April 4, 2018
New Gene Editing Strategy Leads to Much Longer Protein Production
News
American researchers have developed a new way of delivering a gene editing therapy that significantly increases the time a ... Read more

April 20, 2018April 20, 2018

#AAN2018 – Clinical Trials Re-Analysis Shows Emflaza Potential to Preserve Motor Function in Boys with DMD

News

Treatment with Emflaza (deflazacort) is better at preserving motor function in patients with Duchenne muscular dystrophy (DMD) than standard of care corticosteroid treatments, according to a re-analysis of data ... Read more

April 18, 2018April 18, 2018

#AAN2018 — Exondys 51 Found to Be Safe in Young Infant with DMD, Preliminary Data Shows

News

Treatment with Exondys 51 (eteplirsen) was found to be safe and well-tolerated in a very young infant with Duchenne muscular dystrophy (DMD), preliminary data of a follow-up study shows. ... Read more

April 17, 2018April 16, 2018

Noticing the Blessings in Everyday Life

columns, You, Me and MD - a Column by Leah Leilani

Chronic illness can dramatically alter life perspective. If there’s anything I’ve learned from having mitochondrial myopathy, it’s gratitude for my surroundings. I guess I’ve developed a kind of mindfulness ... Read more

April 16, 2018April 16, 2018

UT Researcher, with Cure Duchenne Support, Launches Company to Treat DMD Using CRISPR/Cas9 Technology

News

Eric Olson, a molecular biologist, has spent more than half his life in the lab. Now, the 62-year-old University of Texas researcher and distinguished professor is a businessman as ... Read more

April 13, 2018April 12, 2018

NIH Agency Pioneers Collaborative Research into Rare Diseases

News

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. ... Read more

April 13, 2018April 13, 2018

Pfizer Launches Phase 1b Clinical Trial for Mini-Dystrophin Gene Therapy to Treat DMD

News

Pfizer, Inc began a Phase 1b clinical trial (NCT03362502) for its investigational mini-dystrophin gene therapy product, PF-06939926, in patients with Duchenne muscular dystrophy (DMD). The first dose of the mini-dystrophin ... Read more

April 10, 2018April 9, 2018

Breaking the Stigma of Asking for Help

columns, You, Me and MD - a Column by Leah Leilani

A lot of people think that asking for help is a piece of cake. A walk in the park. But I’ve learned, not only from experience but also from ... Read more

April 10, 2018April 11, 2018

Genetic Compensation Mechanism Could Aid in Treatment of Muscle Disorders, Study Suggests

News

A genetic compensation mechanism discovered in zebra fish could have implications in the treatment of muscle diseases, a recent study suggests. The study, “Genetic compensation triggered by actin mutation ... Read more

April 8, 2018April 9, 2018

PPMD Names PerkinElmer to Conduct Genetic Tests for Its Decode Duchenne Program

News

Parent Project Muscular Dystrophy (PPMD) has selected PerkinElmer to provide genetic testing for its Decode Duchenne program, which offers testing and counseling at no cost to eligible Duchenne muscular dystrophy (DMD) patients and ... Read more

April 5, 2018April 6, 2018

Santhera’s Omigapil Found Safe and Well-Tolerated in Young CMD Patients in Phase 1 Trial

News

Omigapil, an investigational therapy by Santhera Pharmaceuticals, proved to be safe and well-tolerated in children and adolescents with congenital muscular dystrophy (CMD), in a Phase 1 clinical trial. Switzerland-based Santhera ... Read more

April 4, 2018April 4, 2018

Connecticut Neurologist Upbeat on New Therapies for DMD, Other Disorders

News

Gyula Acsadi is an expert in three of the world’s most expensive rare illnesses to treat: Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), and Pompe disease. Acsadi, head of ... Read more

April 4, 2018April 4, 2018

New Gene Editing Strategy Leads to Much Longer Protein Production

News

American researchers have developed a new way of delivering a gene editing therapy that significantly increases the time a repaired gene can generate the protein that’s missing in a ... Read more