AssetMemo

Therapeutic area

Rare & Genetic Diseases

Multisystem monogenic conditions, inborn errors of metabolism, lysosomal storage disorders, and orphan diseases.

Provisional & emerging programs (31)Early clinical pipeline & single-asset indications
Alpha-1 Antitrypsin Deficiency
5 assets
Transthyretin-Mediated Amyloidosis
5 assets· 2 indications
Duchenne Muscular Dystrophy
4 assets
Fabry Disease
4 assets
Myotonic Dystrophy Type 1
3 assets
Phenylketonuria
3 assets
Achondroplasia
2 assets
Facioscapulohumeral Muscular Dystrophy
2 assets
Mucopolysaccharidosis Type II
2 assets
AADC Deficiency
1 asset
Acute Hepatic Porphyria
1 asset
Amyloidosis
1 asset
Angelman Syndrome
1 asset
Bardet-Biedl Syndrome
1 asset
Bronchiolitis Obliterans Syndrome
1 asset
Dystrophic Epidermolysis Bullosa
1 asset
Glycogen Storage Disease Type Ia
1 asset
Hereditary Angioedema
1 asset
Hypochondroplasia
1 asset
Mucopolysaccharidosis Type I
1 asset
Mucopolysaccharidosis Type IIIA
1 asset
Mucopolysaccharidosis Type IVA
1 asset
Mucopolysaccharidosis Type VI
1 asset
Neuronal Ceroid Lipofuscinosis Type 2
1 asset
Osteogenesis Imperfecta
1 asset
Porphyria
1 asset
Prader-Willi Syndrome
1 asset
Primary Hyperoxaluria
1 asset
Rett Syndrome
1 asset
X-linked Adrenoleukodystrophy
1 asset
Ataxia Telangiectasia
0 assets

All assets in Rare & Genetic Diseases

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