Data Support Safety and Bioactivity of Gene Therapy for Pompe Disease
AAV8-LSPhGAA for the treatment of Pompe disease seems to be safe and bioactive, according to results from a phase 1 clinical trial.
MDA Clinical & Scientific Conference 2022
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Diagnostic Delays Affect Quality of Life for Patients With Pompe Disease
Patients with Pompe disease experience a long and distressing diagnostic process and increased isolation and deterioration due to COVID-19.
Phase 1/2 Study Yields Positive Results on AT-GAA for Pompe Disease
Positive results have been announced for a phase 1/2 study on AT-GAA treatment for patients with Pompe disease.
A Better Vector to Deliver Gene Therapy to Muscles?
A new family of adeno-associated virus (AAV) capsid variants enable the delivery of the desired gene specifically to muscles.
Trial Data Support Further Development of Exon-Skipping Drug for DMD
Results from Part A of the phase 2 MOMENTUM trial testing the safety and tolerability of SRP-5051 in Duchenne muscular dystrophy are positive.
MicroRNA miR-128: A Potential New Therapeutic Target for DMD
The microRNA miR-128 is involved in the pathology of Duchenne muscular dystrophy (DMD) and could be a promising approach for therapeutics.
Deflazacort Appears Superior to Prednisone in Delaying Progression of DMD
Deflazacort delays the progression of multiple ambulatory milestones in Duchenne muscular dystrophy (DMD) patients, compared to prednisone.
Three CRISPR-Cas9 Gene Editing Techniques Used to Correct DMD Gene Errors
Gene editing can be effective in correcting mutations in the human genome and treating diseases such as DMD, an expert said at the annual MDA conference.
Factors to Consider When Deciding Between DMD Treatment Options
There are a number of DMD treatment options, with each demonstrating benefits but also potential risks and complications, Katherine Mathews, MD, said.
How Telehealth Can Help Patients With Muscular Dystrophy
Telehealth can help patients with neuromuscular diseases who cannot reach specialized centers, even though the approach has its limitations, experts said.
Positive Interim Results on Casimersen Presented at Annual MDA Conference
Casimersen (Amondys 45) significantly increased dystrophin expression at 48 weeks after treatment initiation in patients with DMD, researchers reported.
SMA Trial Will Evaluate Combination of Risdiplam and Myostatin Antibody
The effect of the sweeping antibody GYM329 in combination with risdiplam in patients with SMA will be investigated in a 2-part clinical trial.
New Analysis Effective in Monitoring Myocardial Involvement in DMD
Assessment of global longitudinal strain can effectively help monitor myocardial involvement in patients with DMD, Brazilian researchers reported.
Nusinersen During Presymptomatic Stage Beneficial for SMA, Data Show
Researchers reported from an ongoing trial that the initiation of nusinersen in infants at the presymptomatic stage of SMA is beneficial.
Growing Data Registries Help Improve Treatment of Neuromuscular Diseases
While more drugs for neuromuscular diseases are being approved than ever before, it’s still a challenge to make those therapies available to all patients.
Pulmonary Hygiene Essential in Preventing Secondary Illness in Patients With DMD
Pulmonary hygiene is essential to preventing illness and reducing hospitalizations rates in patients with neuromuscular disorders including DMD, an expert said at the 2022 Muscular Dystrophy Association Clinical & Scientific Conference.
Novel Modifier Gene May Influence the Severity of Cardiomyopathy in DMD
The level of expression of the MTCH2 gene may be involved in the timing and severity of cardiomyopathy in patients with DMD, according to a new study presented at the 2022 Muscular Dystrophy Association Clinical & Scientific Conference.
Muscular Dystrophy Patients Face Significant Cognitive Learning Challenges
Patients with Duchenne and Becker muscular dystrophies experience high rates of cognitive learning challenges, according to caregiver assessments.
Duchenne Gene Therapy May Have Clinically Relevant Biological Effect
Results from a phase 2 trial suggest delandistrogene moxeparvovec (SRP-9001) has a biological effect that may be clinically relevant in DMD.
Researchers Join Forces to Unravel Cause of Serious Adverse Events in DMD Gene Therapy Trials
Researchers joined forces to better understand the serious adverse events that occurred during trials evaluating gene therapies to treat DMD.
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.
Registration Now Open for MDA’s 2022 Clinical and Scientific Conference
The Muscular Dystrophy Association (MDA)’s 2022 clinical and scientific conference will be held March 13-16 both virtually and in person at the Gaylord Opryland Resort and Convention Center in Nashville, Tennessee. Registration is now open.
Rare Disease Advisor is pleased to offer live coverage of the MDA Clinical & Scientific Conference 2022.
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