Myotonic Dystrophy News and Research

RSS
Protein changes essential for normal adult muscle function

Protein changes essential for normal adult muscle function

New version of gene editing technique corrects molecular mistakes in RNA-based diseases

New version of gene editing technique corrects molecular mistakes in RNA-based diseases

Scientists uncover new gene therapy treatment routes for motor neurone disease

Scientists uncover new gene therapy treatment routes for motor neurone disease

Researchers discover basic genetic mechanism that causes congenital myotonic dystrophy

Researchers discover basic genetic mechanism that causes congenital myotonic dystrophy

TSRI researchers develop new drug candidates to target prostate and triple negative breast cancers

TSRI researchers develop new drug candidates to target prostate and triple negative breast cancers

TSRI scientists develop new methods to design precision medicines against disease-causing RNAs

TSRI scientists develop new methods to design precision medicines against disease-causing RNAs

Tufts scientists discover reason for incidence of genetic error that causes many disorders

Tufts scientists discover reason for incidence of genetic error that causes many disorders

Researchers clarify cause of heart arrhythmia in myotonic dystrophy

Researchers clarify cause of heart arrhythmia in myotonic dystrophy

TSRI scientists develop first drug candidate that neutralizes disease-causing RNA repeats

TSRI scientists develop first drug candidate that neutralizes disease-causing RNA repeats

Biogen begins ISIS-SOD1 Rx Phase 1/2 clinical study in patients with amyotrophic lateral sclerosis

Biogen begins ISIS-SOD1 Rx Phase 1/2 clinical study in patients with amyotrophic lateral sclerosis

Cancer drug can neutralize toxic RNA that causes myotonic dystrophy

Cancer drug can neutralize toxic RNA that causes myotonic dystrophy

Drug compounds target multiple pathways associated with myotonic dystrophy type 1

Drug compounds target multiple pathways associated with myotonic dystrophy type 1

AMO Pharma raises $25 million in private equity financing

AMO Pharma raises $25 million in private equity financing

Isis Pharmaceuticals receives $2.15 million milestone payment to advance ISIS-SMN Rx study in children with SMA

Isis Pharmaceuticals receives $2.15 million milestone payment to advance ISIS-SMN Rx study in children with SMA

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 study in children with SMA

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 study in children with SMA

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 clinical study in infants with Type I SMA

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 clinical study in infants with Type I SMA

UVA researcher identifies potential therapeutic target for myotonic muscular dystrophy

UVA researcher identifies potential therapeutic target for myotonic muscular dystrophy

Isis receives $5 million from Biogen Idec to develop drugs for neurological disorders

Isis receives $5 million from Biogen Idec to develop drugs for neurological disorders

Isis receives $10 million from Biogen Idec to begin IND-supporting studies of ISIS-BIIB4 Rx

Isis receives $10 million from Biogen Idec to begin IND-supporting studies of ISIS-BIIB4 Rx

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients