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OrphanDrug covers global rare drug access intelligence for 193 countries.

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Drug Database

284 entries
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Orphan Drugs Only
Ivosidenib
Orphan14dFDA Approved
Ketoconazole
FDA Approved
L-carnitine
Orphan5dFDA Approved, EMA Approved
L-DOPA
FDA Approved
L-Methylfolate
5dFDA Approved
Lanreotide
Orphan Cold Chain14dFDA Approved
laronidase
Orphan Cold Chain14dFDA Approved, EMA Approved
Laser photocoagulation therapy
OrphanFDA Approved
Levetiracetam
3dFDA Approved, EMA Approved
Levodopa/Carbidopa
7dFDA Approved
Levothyroxine
FDA Approved
levothyroxine sodium
3dFDA Approved
Lomitapide
OrphanFDA Approved
lonafarnib
Orphan21dFDA Approved, EMA Approved
loncastuximab tesirine
Orphan Cold Chain14dFDA Approved (2021), EMA Approved (2022)
Lorenzo's oil
Lumasiran
Orphan Cold ChainFDA Approved, EMA Approved
luspatercept
Orphan Cold Chain10dFDA Approved, EMA Approved
mannose
Orphan14dInvestigational
mannose-1-phosphate
Orphan Cold Chain30dInvestigational
mavacamten
Orphan14dFDA Approved
mecasermin
Orphan Cold Chain14dFDA Approved, EMA Approved
melatonin
Orphan7dEMA Approved, FDA OTC
Melphalan
7dFDA Approved
Memantine
5dFDA Approved
Mesalamine
7dFDA Approved
metformin
2dFDA Approved
Methotrexate
Orphan3dFDA Approved
metoprolol
2dFDA Approved
Metreleptin
Orphan Cold Chain21dFDA Approved
Page 6 of 10 (284 total)
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