Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.
Novel gene therapy for diseased muscle fiber repair

Novel gene therapy for diseased muscle fiber repair

New findings could contribute to future therapeutics for muscle degeneration

New findings could contribute to future therapeutics for muscle degeneration

New gene therapy addresses primary cellular deficit associated with limb-girdle muscular dystrophy 2B

New gene therapy addresses primary cellular deficit associated with limb-girdle muscular dystrophy 2B

Single-patient pilot study shows the ability of antisense oligonucleotide to suppress mutant ALS gene

Single-patient pilot study shows the ability of antisense oligonucleotide to suppress mutant ALS gene

New consensus-based recommendations designed to improve quality of life for DMD patients

New consensus-based recommendations designed to improve quality of life for DMD patients

First pediatric-only guidelines for the use of pacemakers and implantable defibrillators

First pediatric-only guidelines for the use of pacemakers and implantable defibrillators

Study shows long-term safety and biologic activity of golodirsen in DMD patients

Study shows long-term safety and biologic activity of golodirsen in DMD patients

Study offers new insight into the role of tau proteins in ALS pathogenesis

Study offers new insight into the role of tau proteins in ALS pathogenesis

Lamin C important for keeping cell’s genetic material organized, research shows

Lamin C important for keeping cell’s genetic material organized, research shows

New therapy harnesses "dancing molecules" to reverse paralysis, repair tissue after spinal cord injuries

New therapy harnesses "dancing molecules" to reverse paralysis, repair tissue after spinal cord injuries

Health care costs for people with rare diseases have been underestimated, study shows

Health care costs for people with rare diseases have been underestimated, study shows

Researchers illustrate the importance and value of sharing negative research results

Researchers illustrate the importance and value of sharing negative research results

Parent Project Muscular Dystrophy successfully completes the Newborn Screening Pilot project

Parent Project Muscular Dystrophy successfully completes the Newborn Screening Pilot project

Specialized cells that keep the heart beating have ability to regenerate after birth, study reveals

Specialized cells that keep the heart beating have ability to regenerate after birth, study reveals

Early treatment with ACE inhibitors improves cardiac outcomes of patients with Becker muscular dystrophy

Early treatment with ACE inhibitors improves cardiac outcomes of patients with Becker muscular dystrophy

Newly developed antibody may be used to treat muscular dystrophy

Newly developed antibody may be used to treat muscular dystrophy

New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases

New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases

Pfizer COVID-19 jab side effects likely to be mild to moderate in extremely vulnerable 12-15 year olds

Pfizer COVID-19 jab side effects likely to be mild to moderate in extremely vulnerable 12-15 year olds

Research could enable clinicians to produce patient-matched cells for treating muscle-related diseases

Research could enable clinicians to produce patient-matched cells for treating muscle-related diseases

Telehealth helped physical therapists to provide essential care for pediatric patients during pandemic

Telehealth helped physical therapists to provide essential care for pediatric patients during pandemic