Posts by: Iqra Mumal

News

Wave Life Sciences’ treatment candidate WVE-210201 has been granted both orphan drug designation and rare pediatric disease designation for the treatment of Duchenne muscular dystrophy (DMD) from the U.S. Food ... 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

December 19, 2017December 19, 2017

Gene Therapy Approach Using HAC Vector May Work with Dystrophin Gene, Study Suggests

News

A new way of transferring the dystrophin gene — the largest known human gene — into muscle stem cells may be possible using the human artificial chromosome (HAC) vector, a ... Read more

December 18, 2017December 18, 2017

Problems in IL-6 Pathway Linked to Severe Symptoms of Congenital Myotonic Dystrophy in Study

News

Aberrant signaling in the IL-6 pathway, which is involved in muscle cell maturation, contributes to the severe symptoms seen in congenital myotonic dystrophy (CDM) patients, researchers in Japan report. The ... Read more

December 15, 2017December 15, 2017

Investigational Therapy SR8278 Shown to Regenerate Muscle in DMD Mice Study

News

Scientists at Saint Louis University believe that an investigational therapy called SR8278 holds potential for treating Duchenne muscular dystrophy (DMD) after it was shown to increase muscle function and decrease ... Read more

December 13, 2017

Muscle MRI Can Predict Functional Deterioration in Patients with Becker MD, Study Says

News

Researchers have found that skeletal muscle magnetic resonance imaging (MRI) correlates with motor function and can help predict the degree of functional deterioration in patients with Becker muscular dystrophy ... Read more

November 14, 2017

Protein Utrophin Reduces Mitochondria Damage in Duchenne MD, Study Shows

News

In recent years scientists have learned that the protein utrophin can substitute for the dystrophin protein that is missing in muscular dystrophy. Researchers have now discovered that utrophin can reduce ... Read more

November 9, 2017

Wave Begins Phase 1 Trial of Therapy That Addresses Exon Skipping in Duchenne MD

News

Wave Life Sciences has started a Phase 1 clinical trial of a treatment for a gene malfunction linked to the most common form of Duchenne muscular dystrophy. Genes consists of dozens ... Read more

November 1, 2017

Sarepta and Duke University Collaborating on Gene Editing as a Treatment for Duchenne MD

News

Sarepta Therapeutics is working with Duke University’s biomedical engineering program program on gene editing therapies for Duchenne muscular dystrophy. The editing approach the partners are using is called CRISPR/Cas9. It can correct mutations by removing ... Read more

Posts by: Iqra Mumal

Iqra Mumal

WVE-210201 Receives FDA Orphan Drug, Rare Pediatric Disease Designations for DMD

New Gene Editing Strategy Leads to Much Longer Protein Production

Gene Therapy Approach Using HAC Vector May Work with Dystrophin Gene, Study Suggests

Problems in IL-6 Pathway Linked to Severe Symptoms of Congenital Myotonic Dystrophy in Study

Investigational Therapy SR8278 Shown to Regenerate Muscle in DMD Mice Study

Muscle MRI Can Predict Functional Deterioration in Patients with Becker MD, Study Says

Protein Utrophin Reduces Mitochondria Damage in Duchenne MD, Study Shows

Wave Begins Phase 1 Trial of Therapy That Addresses Exon Skipping in Duchenne MD

Sarepta and Duke University Collaborating on Gene Editing as a Treatment for Duchenne MD

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