Thiago Carvalho @cyrilpedia@qoto.org

My Clinical Pipeline column for Nature Medicine this week examines the state of Sarepta's new gene therapy for Duchenne muscular dystrophy application at the FDA. Whatever its final efficacy turns out to be, the general strategy behind the therapy, an engineered micro-dystrophin is very clever.

It was inspired by the case of a 61 year old patient who had a deletion of almost half of his dystrophin gene and was still alive and walking. Here's that ref, from 1990:
https://www.nature.com/articles/343180a0

And here's my column:

https://www.nature.com/articles/d41591-023-00052-4

"As of January 2023, the estimated prevalence of long Covid among people in the United States who had had acute infection was 11%. According to guidance released in August 2021 by the Department of Health and Human Services and the Department of Justice, long Covid can be considered a protected disability under the Americans with Disabilities Act (ADA) and under other disability-antidiscrimination mandates (such as Section 504 of the Rehabilitation Act and Section 1557 of the Affordable Care Act)."

#covid #longcovid #covid19

https://www.nejm.org/doi/full/10.1056/NEJMp2302676?query=featured_home

'Here, we generated a mouse model with insufficient ketogenesis by conditionally knocking out the gene encoding the hepatocyte-specific ketogenic enzyme hydroxymethylglutary-CoA synthase 2 (Hmgcs2 KO). Intriguingly, erythroid maturation was enhanced with boosted fatty acid synthesis in bone marrow of hepatic Hmgcs2 KO mouse under fasting condition, suggesting that systemic ketogenesis has a profound effect on erythropoiesis.'

#Preprint #metabolism

https://www.biorxiv.org/content/10.1101/2023.06.07.544102v1

'O’Donnell’s plan came good: Lumumba was captured on the road by Mobutu’s officers and flown to Katanga, where he was murdered within hours. The CIA’s broad agenda for the DRC was supported, Williams writes, ‘by the highest level of the government’. The so-called Special Group, a subcommittee of the National Security Council responsible for covert operations, had given the CIA station in Léopoldville the green light and wired Devlin $100,000 to finish the job.'

https://www.lrb.co.uk/the-paper/v45/n12/kevin-okoth/poison-is-better

'Using an industry-standard test, he measured the particulate matter outside the mask, then checked how much made it through the device and into the space around his nose and mouth. Percentage-wise, he told me, “it removes 99.99 … I didn’t measure how many nines; it was working so well.” On broader scales, too, the protective math plays out: Well-fitting masks can curb smoke-related hospitalizations; studies back up their importance as a firefighting mainstay.'

https://www.theatlantic.com/health/archive/2023/06/mask-wildfire-smoke-n95-effective/674325/

'Los investigadores sufren una presión brutal para publicar estudios. Sus aumentos de sueldo, sus ascensos, la financiación de sus proyectos y su reconocimiento social dependen de evaluaciones en las que su rendimiento se mide prácticamente al peso. Este sistema, conocido como “publica o muere”, ha creado monstruos.'
https://elpais.com/ciencia/2023-06-03/un-cientifico-que-publica-un-estudio-cada-dos-dias-muestra-el-lado-mas-oscuro-de-la-ciencia.html

'Several pieces of EU legislation in sectors including banking and pharmaceuticals face severe delays because it is taking too long to translate documents, the European parliament has said.'

https://www.ft.com/content/024e7faa-28dc-4332-b6ae-9e60f7daf4e2

'Weight loss supplements are among the most likely supplements to be adulterated and potentially contaminated with banned substances, Dr. Cohen said. Data from the Centers for Disease Control and Prevention shows that herbal products marketed for weight loss are the type of supplements that most frequently send people to the emergency room, with patients often reporting adverse effects like palpitations or chest pain.'

https://www.nytimes.com/2023/06/07/well/eat/berberine-weight-loss-ozempic.html?action=click&module=Well&pgtype=Homepage&section=Well

‘In the winter of 1750, Jean-Jacques Rousseau threw away his watch. “Thank heavens,” he remembered exclaiming after this sublime gesture, “I shall no longer need to know what time its!”’

- Peter Gay, ‘The Enlightenment, the rise of modern paganism’.

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