Top 13 dmd treatment in 2022

Below are the best information and knowledge on the subject dmd treatment compiled and compiled by our own team alltopus:

1. Drug treatment of Duchenne muscular dystrophy: available evidence and perspectives – PMC

Author: stanfordhealthcare.org

Date Submitted: 04/17/2021 06:05 PM

Average star voting: 4 ⭐ ( 33938 reviews)

Summary:

Match with the search results: Diet and nutrition counseling for patients with difficulty chewing or swallowing….. read more

Drug treatment of Duchenne muscular dystrophy: available evidence and perspectives - PMC

2. Combined Therapies for Duchenne Muscular Dystrophy to Optimize Treatment Efficacy

Author: www.mda.org

Date Submitted: 04/22/2019 11:14 AM

Average star voting: 5 ⭐ ( 74825 reviews)

Summary: Duchene Muscular Dystrophy (DMD) is the most frequent muscular dystrophy and one of the most severe due to the absence of the dystrophin protein. Typical pathological features include muscle weakness, muscle wasting, degeneration and inflammation. At advanced stages DMD muscles present exacerbated extracellular matrix and fat accumulation. Recent progress in therapeutic approaches has allowed new strategies to be investigated, including pharmacological, gene-based and cell-based therapies. Gene and cell-based therapies are still limited by poor targeting and low efficiency in fibrotic dystrophic muscle, therefore it is increasingly evident that future treatments will have to include “combined therapies” to reach maximal efficiency. The scope of this mini-review is to provide an overview of the current literature on such combined therapies for DMD. By “combined therapies” we mean those that include both a therapy to correct the genetic defect and an additional one to address one of the secondary pathological features of the disease. In this mini-review, we will not provide a comprehensive view of the literature on therapies for DMD, since many such reviews already exist, but we will focus on the characteristics, efficiency and potential of such combined therapeutic strategies that have been described so far for DMD.

Match with the search results: The corticosteroids prednisone and deflazacort are beneficial in the treatment of DMD. The FDA on Feb. 9, 2017, approved deflazacort (brand name Emflaza), an ……. read more

Combined Therapies for Duchenne Muscular Dystrophy to Optimize Treatment Efficacy

3. What Are the Best Treatments for Duchenne Muscular Dystrophy (DMD)?

Author: www.webmd.com

Date Submitted: 09/03/2019 05:47 AM

Average star voting: 5 ⭐ ( 80218 reviews)

Summary: Steroids, along with physical therapy and appropriate drugs and devices for heart and breathing problems, form the core of treatment for DMD.

Match with the search results: There’s no cure for DMD, but there are medicines and other therapies that can ease your child’s symptoms, protect their muscles, and keep their ……. read more

What Are the Best Treatments for Duchenne Muscular Dystrophy (DMD)?

4. Duchenne Muscular Dystrophy – TREAT-NMD

Author: rarediseases.info.nih.gov

Date Submitted: 04/24/2020 11:59 PM

Average star voting: 5 ⭐ ( 78856 reviews)

Summary:

Match with the search results: There is no known cure for Duchenne muscular dystrophy (DMD). Treatment is focused on managing the symptoms of DMD and related complications ……. read more

Duchenne Muscular Dystrophy - TREAT-NMD

5. Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy | European Journal of Human Genetics

Author: www.ncbi.nlm.nih.gov

Date Submitted: 02/08/2019 04:57 PM

Average star voting: 4 ⭐ ( 69585 reviews)

Summary: Duchenne muscular dystrophy (DMD) is an X-linked progressive muscle-wasting disorder that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the structural integrity of muscle. As variants in the dystrophin gene lead to a disruption of the reading frame, pharmacological treatments have only limited efficacy; there is currently no effective therapy and consequently, a significant unmet clinical need for DMD. Recently, novel genetic approaches have shown real promise in treating DMD, with advancements in the efficacy and tropism of exon skipping and surrogate gene therapy. CRISPR-Cas9 has the potential to be a ‘one-hit’ curative treatment in the coming decade. The current limitations of gene editing, such as off-target effects and immunogenicity, are in fact partly constraints of the delivery method itself, and thus research focus has shifted to improving the viral vector. In order to halt the loss of ambulation, early diagnosis and treatment will be pivotal. In an era where genetic sequencing is increasingly utilised in the clinic, genetic therapies will play a progressively central role in DMD therapy. This review delineates the relative merits of cutting-edge genetic approaches, as well as the challenges that still need to be overcome before they become clinically viable.

Match with the search results: Glucocorticoids, more precisely prednisone and deflazacort, are the main drug treatment for DMD. They have been used for over two decades and the benefits are ……. read more

Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy | European Journal of Human Genetics

6. Experimental Gene Therapy Targets Duchenne Muscular Dystrophy

Author: www.fda.gov

Date Submitted: 11/27/2019 07:56 AM

Average star voting: 5 ⭐ ( 31748 reviews)

Summary: Children in Rochester were recently among the first in the nation to be enrolled in a phase 3 clinical trial for a new gene therapy to treat Duchenne muscular dystrophy (DMD).

Match with the search results: FDA has granted accelerated approval to Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy in patients who ……. read more

Experimental Gene Therapy Targets Duchenne Muscular Dystrophy

7. New ‘cocktail’ drug could benefit up to 45 per cent of patients with Duchenne muscular dystrophy

Author: medlineplus.gov

Date Submitted: 10/21/2021 08:09 PM

Average star voting: 3 ⭐ ( 94284 reviews)

Summary: A new ‘cocktail’ drug under development could provide an effective and economical treatment to lessen symptoms for up to 45 per cent of patients with Duchenne muscular dystrophy (DMD), a chronic muscle-wasting disease.

Match with the search results: Treatment · Assisted ventilation (used during the day or night) · Drugs to help heart function, such as angiotensin converting enzyme inhibitors, ……. read more

New 'cocktail' drug could benefit up to 45 per cent of patients with Duchenne muscular dystrophy

8. Duchenne Muscular Dystrophy in Children | Cedars-Sinai

Author: www.mayoclinic.org

Date Submitted: 12/18/2019 09:29 PM

Average star voting: 3 ⭐ ( 42046 reviews)

Summary: Duchenne muscular dystrophy is a rare genetic condition that weakens your child’s
muscles. It appears in young boys, usually between ages 2 and 5.

Match with the search results: Treatment options include medications, physical and occupational therapy, … to treat some people with Duchenne muscular dystrophy….. read more

Duchenne Muscular Dystrophy in Children | Cedars-Sinai

9. CRISPR technologies for the treatment of Duchenne muscular dystrophy

Author: rarediseases.org

Date Submitted: 10/21/2019 02:36 PM

Average star voting: 4 ⭐ ( 92004 reviews)

Summary:

Match with the search results: Duchenne muscular dystrophy (DMD) is a rare muscle disorder but it is one of … In 2016, Exondys 51 (eteplirsen) injection was FDA approved to treat DMD ……. read more

CRISPR technologies for the treatment of Duchenne muscular dystrophy

10. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review – Orphanet Journal of Rare Diseases

Author: www.hopkinsmedicine.org

Date Submitted: 02/23/2021 11:26 AM

Average star voting: 4 ⭐ ( 68898 reviews)

Summary: Duchenne Muscular Dystrophy (DMD) is a rapidly progressive, lethal neuromuscular disorder, present from birth, which occurs almost exclusively in males. We have reviewed contemporary evidence of burden, epidemiology, illness costs and treatment patterns of DMD. This systematic review adhered to published methods with information also sought from the web and contacting registries. Searches were carried out from 2005 to June 2015. The population of interest was individuals with clearly defined DMD or their carers. Nine thousand eight hundred fifty titles were retrieved from searches. Fifty-eight studies were reviewed with three assessed as high, 33 as medium and 22 as low quality. We found two studies reporting birth and four reporting point prevalence, three reporting mortality, 41 reporting severity and/or progression, 18 reporting treatment patterns, 12 reporting quality of life, two reporting utility measures, three reporting costs of illness and three treatment guidelines. Birth prevalence ranged from 15.9 to 19.5 per 100,000 live births. Point prevalence per 100,000 males was for France, USA, UK and Canada, 10.9, 1.9, 2.2 and 6.1 respectively. A study of adult DMD patients at a centre in France found median survival for those born between 1970 and 1994 was 40.95 years compared to 25.77 years for those born between 1955 and 1969. Loss of ambulation occurred at a median age of 12 and ventilation starts at about 20 years. There was international variation in use of corticosteroids, scoliosis surgery, ventilation and physiotherapy. The economic cost of DMD climbs dramatically with disease progression – rising as much as 5.7 fold from the early ambulatory phase to the non-ambulatory phase in Germany. This is the first systematic review of treatment, progression, severity and quality of life in DMD. It also provides the most recent description of the burden, epidemiology, illness costs and treatment patterns in DMD. There are evidence gaps, particularly in prevalence and mortality. People with DMD seem to be living longer, possibly due to corticosteroid use, cardiac medical management and ventilation. Future research should incorporate registry data to improve comparability across time and between countries and to investigate the quality of life impact as the condition progresses.

Match with the search results: A gene therapy clinical trial may open up new possibilities for the treatment of Duchenne muscular dystrophy….. read more

The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review - Orphanet Journal of Rare Diseases

11. Treatment for Duchenne muscular dystrophy mutation approved by FDA

Author: www.frontiersin.org

Date Submitted: 02/21/2020 01:42 PM

Average star voting: 5 ⭐ ( 14782 reviews)

Summary: The FDA conditionally approved Amondys 45 (casimersen) after interim Phase III results indicated it is likely to be of clinical benefit.

Match with the search results: Diet and nutrition counseling for patients with difficulty chewing or swallowing….. read more

Treatment for Duchenne muscular dystrophy mutation approved by FDA

12. Eteplirsen treatment for Duchenne muscular dystrophy

Author: www.frontiersin.org

Date Submitted: 05/01/2019 10:00 PM

Average star voting: 3 ⭐ ( 90875 reviews)

Summary:

Match with the search results: The corticosteroids prednisone and deflazacort are beneficial in the treatment of DMD. The FDA on Feb. 9, 2017, approved deflazacort (brand name Emflaza), an ……. read more

Eteplirsen treatment for Duchenne muscular dystrophy

13. Duchenne Muscular Dystrophy (DMD) Treatment: Past and Present Perspectives | IntechOpen

Author: www.everydayhealth.com

Date Submitted: 07/19/2020 04:57 AM

Average star voting: 5 ⭐ ( 22334 reviews)

Summary: Duchenne muscular dystrophy (DMD) is one of the fatal X-linked disorders that are characterized by progressive muscle weakness and occur due to mutation in the largest human gene known as the DMD gene

Match with the search results: There’s no cure for DMD, but there are medicines and other therapies that can ease your child’s symptoms, protect their muscles, and keep their ……. read more

Duchenne Muscular Dystrophy (DMD) Treatment: Past and Present Perspectives | IntechOpen

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