Top 5 Most Read Spinal Muscular Atrophy Stories of 2021

The 5 most-read spinal muscular atrophy stories of 2021 on AJMC.com covered a variety of topics regarding the disease, including cost differences, the potential for combination therapies, and the identification of biomarkers for treatment.

The most clicked pieces of content in 2021 that dealt with spinal muscular atrophy (SMA) covered a variety of topics regarding the disease, including some articles that addressed metabolic and nutritional issues associated with SMA, as well as how to ” improve results-based management. entry agreements.

Other articles have discussed the future of combination therapies for SMA and how the costs of managing SMA differ around the world.

5. SMA associated with metabolic and nutritional problems

Researchers analyzed data on metabolic and nutritional problems associated with ADS, concluding that the impact of impaired motor neuron survival may extend beyond motor neurons. Some of the documented problems that were reported included lipid metabolic abnormalities, glucose metabolic abnormalities, and altered vitamin levels. Several fatty acids and serum lipids have been identified to correlate with motor function in ADS and could potentially be used as biomarkers for treatment.

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4. Improve Outcome-Based Managed Entry Agreements for SMA Therapy

The results of the study showed that innovative therapies for rare diseases, including ADS, when funded by public budgets in the European Union and elsewhere, are often managed through agreements concluded by payers and drug companies. However, these results-based managed entry agreements can be difficult to implement and should be seen as an exception rather than a rule. Information on these agreements can also be difficult to find, sometimes due to the confidential business information that is involved.

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3. Dr Vamshi Rao discusses the future of combination therapy in ADS

Vamshi Rao, MD, attending physician in neurology at Ann and Robert H. Lurie Children’s Hospital in Chicago and assistant professor of pediatrics (neurology and epilepsy) at Northwestern University Feinberg School of Medicine, spoke about the future of combination therapy in SMA. space. With 3 therapies approved, the surge and interest in combination therapy generally increased. Rao also discussed his hopes for more concrete evidence of its use.

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2. Researchers highlight emerging concepts underlying selective neuromuscular dysfunction in ADS

A group of researchers presented the available evidence of emerging molecular and cellular mechanisms that have been implicated in selective neuromuscular dysfunction in infants with ADS. The 25-year data review highlighted the importance of research into emerging concepts, which may offer insight into the success of AMS treatments, and demonstrated why proximally innervated motor neurons are selectively vulnerable. in AMS.

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1. The costs of ADS vary widely around the world depending on the type of disease.

The results of the study showed that reliable data on the costs associated with SMA is scarce and that key questions remain unanswered as expensive therapies continue to change the landscape of SMA treatment. The researchers found only 14 studies from 8 countries, which revealed a wide range of costs depending on the disease phenotype and the country in which the study was conducted, ranging from $ 3,320 in Italy to $ 324,210. $ in the United States.

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