Exciting breakthroughs in rare disease treatments mean more patients are gaining access to cutting-edge therapies - and specialty pharmacies are playing a crucial role in delivering these medications with expert support. But what exactly do they do, and when do they come into play?
AllMyHealth’s latest guide breaks it all down - exploring how specialty pharmacies work, their expanding role in patient care, and how individuals can engage with them. Plus, we spotlight some specialty pharmacies that cater to Huntington's.
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In the realm of Huntington's disease (HD) treatment, Mukherjee et al. (2025) discuss the potential of immunotherapy, a method that utilizes the body's immune system to combat disease. Specifically, the review focuses on strategies that target the mutant huntingtin and tau proteins, which are implicated in the neurodegenerative process of HD. By neutralizing these toxic proteins, the therapies aim to reduce the damage to brain cells. The researchers highlight that targeting sequences flanking the polyglutamine tract in the mutant huntingtin protein, rather than the tract itself, has shown promising results in alleviating symptoms.
However, the review also outlines significant challenges, such as the difficulty in delivering therapeutic agents across the blood-brain barrier, a protective shield that prevents many drugs from entering the brain. Mukherjee et al. emphasize the need for ongoing research to develop effective strategies for breaching this barrier. Despite these hurdles, the repurposing of immunotherapeutic strategies designed for other neurodegenerative diseases offers a hopeful avenue for HD treatment, suggesting that advancements in this field could be beneficial for patients with HD.
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