Remove tag neuromuscular-diseases
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PharmaShots Weekly Snapshots (May 15 - 19, 2023)

PharmaShots

Alimera Acquires Rights from EyePoint Pharmaceuticals to Commercialise Yutiq in the US Date: May 19, 2023 | Tags: Alimera, EyePoint Pharmaceuticals, Yutiq, Iluvien, chronic non-infectious uveitis, US, Pharma Sobi Reports EMA’s Validation of MAA for Efanesoctocog Alfa to Treat Haemophilia A Date: May 19, 2023 | Tags: Sobi, Efanesoctocog Alfa, (..)

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PharmaShots Weekly Snapshots (January 23 - 27, 2023)

PharmaShots

Magenta Therapeutics Pauses the P-I/II Study in AML Patients Date: Jan 27, 2023 | Tags: Magenta Therapeutics, MGTA-117, AML, Clinical Trial, P-I/II Ipsen Receives CHMP Negative Opinion for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva Date: Jan 27, 2023 | Tags: Ipsen, Palovarotene, Fibrodysplasia Ossificans Progressiva, Regulatory, CHMP, (..)

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NICE ‘no’ to Roche’s risdiplam for SMA not unexpected, says patient group

pharmaphorum

The official statement from NICE on the rejection said that its appraisal committee was convinced by Roche’s data showing that Evrysdi improves motor function in people with type 1, 2 or 3 SMA, a progressive neuromuscular condition that can lead to muscle weakness, loss of movement, and difficulty breathing and swallowing.

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Hydrocodone acetaminophen alternatives: What can I take instead of hydrocodone acetaminophen?

The Checkup by Singlecare

Opioids should not be used as first-line therapy for short-term or chronic pain —with the exception being in end-of-life or palliative care, during active cancer treatment, and with sickle cell disease. Belbuca is a good alternative to opioid treatment that needs to be taken around the clock, but it may come with a hefty price tag.

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Realising milestones with gene therapy for SMA

European Pharmaceutical Review

Rare, genetic diseases like SMA are prime targets for gene therapy, but by their very nature, early signs can be difficult to recognise, delaying diagnosis and treatment. 1 This is assuming a $1M price tag for all gene therapies. In reality, this will vary by disease area and therapy.

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Tramadol (Ultram) alternatives: What can I take instead of tramadol?

The Checkup by Singlecare

The Centers for Disease Control and Prevention (CDC) have committed to fighting the opioid overdose epidemic in many ways, including by aiding providers, healthcare systems, and payers with data, tools, and evidence-based guidance to support improved opioid prescribing and patient safety. fibromyalgia ).