nizubaglustat

Indicaton

Has the potential to treat multiple rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses, and Niemann-Pick disease type C (NPC).

Therapeutic indications

Type of product

Small molecule

Table

Phase

Phase II started
Partnering status

Not partnered
Available in

Global

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Siklos (hydroxyurea)

Siklos is indicated for the prevention of recurrent painful vaso-occlusive crises including acute chest syndrome in adults, adolescents and children older than 2 years suffering from symptomatic sickle cell syndrome

Filing

3D CoSeedis in chip Assays

Preclinical 3D Study models for oncology and neurology.

Marketed

Bimiralisib, topical [PQR309] (panPI3K/mTOR)

Non-melanoma skin cancers and rare genetic skin manifestations.

Discovery

nizubaglustat

Indicaton

Therapeutic indications

Type of product

Company

Table

You may also be interested in

Siklos (hydroxyurea)

3D CoSeedis in chip Assays

Bimiralisib, topical [PQR309] (panPI3K/mTOR)