A Small Molecule for a Large Disease  

Oleh:

Pyeritz, Reed E.

Jenis: Article from Journal - ilmiah internasional
Dalam koleksi: The New England Journal of Medicine (keterangan: ada di Proquest) vol. 358 no. 26 (Jun. 2008), page 2829.

Ketersediaan

Perpustakaan FK Nomor Panggil: N08.K.2008.03 Non-tandon: 1 (dapat dipinjam: 0) Tandon: tidak ada

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Isi artikel

Almost three decades ago, Victor McKusick and I reviewed Marfan's syndrome in the Journal and advised traditional medical and surgical approaches to management.1 The holy grail at that time was the cause of this autosomal dominant condition, based on the common presumption that understanding the cause would lead directly to effective therapy. In fact, life expectancy improved dramatically in subsequent years for all but the most severely affected patients, despite a lack of understanding of the underlying connective-tissue defect.2 Dissection of the aorta was and remains the most common cause of death. However, the evolution of ever more effective surgical . . .

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