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Can the FDA keep the momentum going for rare disease drug approvals?

Pharmaceutical Technology

The mutation leads to disordered thyroid hormone transport, which causes patients to experience intellectual and motor disabilities. Egetis is banking on tiratricol, an oral small molecule that restores dysfunctional thyroid hormone signalling, to be the first drug approved for this condition. In clinical trials, the drug showed a 3.8-point

Drugs 317
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Povorcitinib Phase III Results Stir Mixed Signals for Incyte and Investors

XTalks

Incytes recent announcement on the topline results from two Phase III clinical trials of povorcitinib in patients with hidradenitis suppurativa has stirred both hope and caution in the market. By inhibiting this protein, povorcitinib reduces the inflammatory signals that contribute to abscess and nodule formation.

Trials 59
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Protein Precision: “Harnessing Targeted Protein Degradation”

Roots Analysis

Proteins are the chains of amino acids linked together in different combinations and perform a wide variety of functions. However, continuous regulation of the cellular proteome is essential for maintaining the right balance of different proteins necessary for normal cellular function, survival, and proliferation.

Protein 40
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ARVINAS AND PFIZER ANNOUNCE GLOBAL COLLABORATION TO DEVELOP AND COMMERCIALIZE PROTAC® PROTEIN DEGRADER ARV-471

The Pharma Data

(NYSE: PFE) today announced a global collaboration to develop and commercialize ARV-471, an investigational oral PROTAC® (PROteolysis TArgeting Chimera) estrogen receptor protein degrader. ER is the primary driver of hormone receptor (HR) positive breast cancer, which is the most common breast cancer subtype.

Protein 52
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International Women’s Day 2024: A Spotlight on 5 Women’s Health Webinars with Xtalks

XTalks

Optimizing Sleep During Perimenopause and Beyond Sleep plays a crucial role in overall health, particularly for midlife women transitioning into menopause, a phase marked by perimenopause where hormonal changes can severely disrupt sleep due to symptoms like nocturnal hot flashes.

Genetics 115
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Why the Potential Effects of COVID-19 Vaccines on the Menstrual Cycle Are Not Concerning

XTalks

Currently, there is no scientific evidence to suggest that the vaccines induce any hormonal changes that would lead to irregular periods. However, the similarity between the two proteins is not great enough for anti-spike antibodies to cross-react with placental syncytin-1.

Vaccine 105
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Lumos Pharma’s Patient-Centric Strategy for Rare Disease Drug Development

XTalks

Pediatric growth hormone deficiency (PGHD) is one such condition, affecting a child’s growth and development. This rare endocrine disorder, characterized by inadequate secretion of growth hormone from the pituitary gland, has profound impacts on physical and sometimes cognitive development.