Healthcare companies dealing with rare diseases are significant for investors. These companies have a direct and favorable regulatory pathway for drug approval, and once approved the fear of generics or competition becomes less. Synageva Biopharma (GEVA) is a good example of this. Its lead pipeline candidate, Sebelipase Alfa, is in Phase III development for lysosomal acid lipase, or LAL, deficiency. I see Sebelipase Alfa as a low risk development applicant with a $600 million market potential. Sebelipase Alfa, along with a broad pipeline of other applicants for rare diseases, makes Synageva an attractive investment for long-term growth.
Rare and ultra-rare diseases like Lysosomal Storage Disorders, or LSDs, Mucopolysaccharidosis, or MPS, and TTR-mediated amyloidosis, or ATTR, generally...
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