rare disease
NHS signs deal with Orchard for pricey Libmeldy gene therapy
gene therapy, HEOR, Libmeldy, NHS, NICE, Orchard Therapeutics, paediatric, rare disease
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Pharming preps filing for rare disease drug leniolisib after trial win
activated PI3K delta syndrome, leniolisib, Novartis, Pharming, rare disease
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After FDA setback, Ipsen gets first OK for rare disease drug palovarotene
Canada, garetosmab, Ipsen, Orphan drug, rare disease, Regeneron, Sohonos
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Sarepta says early filing for DMD gene therapy may be back on
Duchenne muscular dystrophy, gene therapy, Pfizer, rare disease, Sarepta, Solid Biosciences
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Regeneron hands ex-US rights to HoFH drug Evkeeza to Ultragenyx
Evkeeza, homozygous familial hypercholesterolaemia, rare disease, Regeneron, Sales and Marketing, Ultragenyx
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Biogen looks beyond Spinraza in SMA with Ionis deal
Biogen, Evrysdi, Ionis Pharmaceuticals, Novartis, rare disease, Roche, spinal muscular atrophy, Spinraza, Zolgensma
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Applied Therapeutics slumps as FDA seeks more data on lead drug
Applied Therapeutics, galactosaemia, inherited disease, rare disease, regulatory
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