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Rare Pediatric Disease

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November 13, 2020
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Vamorolone demonstrates efficacy, safety for boys with Duchenne muscular dystrophy

Vamorolone demonstrates efficacy, safety for boys with Duchenne muscular dystrophy

Treatment with vamorolone, an anti-inflammatory steroidal drug, improved motor outcomes in boys with Duchenne muscular dystrophy compared with corticosteroid-naive individuals, according to findings published in PLoS Medicine.

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September 26, 2020
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Innova Therapeutics receives rare pediatric disease designation for osteosarcoma treatment

Innova Therapeutics Inc. has been granted rare pediatric disease designation by the FDA for its IVT-8086 osteosarcoma treatment.

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August 26, 2020
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FDA grants rare pediatric disease designation to cell therapy for sickle cell disease

FDA grants rare pediatric disease designation to cell therapy for sickle cell disease

The FDA granted rare pediatric disease designation to EDIT-301, an investigational autologous cell therapy for the treatment of sickle cell disease, according to the agent’s manufacturer.

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January 31, 2020
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FDA grants rare pediatric disease designation for QR-421a in retinitis pigmentosa

QR-421a has received rare pediatric disease designation from the FDA for the treatment of retinitis pigmentosa, according to a press release from ProQR Therapeutics.

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January 10, 2020
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FDA grants rare pediatric disease designation to gene therapy for sickle cell disease

The FDA granted rare pediatric disease designation to ARU-1801, an investigational autologous gene therapy, for the treatment of sickle cell disease, according to the agent’s manufacturer.

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October 23, 2019
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ProQR announces rare pediatric disease designation, phase 1/2 results for sepofarsen

Sepofarsen has received rare pediatric disease designation from the FDA for the treatment of Leber’s congenital amaurosis 10, according to a press release from ProQR Therapeutics.

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October 21, 2019
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Triple therapy approved to treat cystic fibrosis

Triple therapy approved to treat cystic fibrosis

The FDA announced it has approved elexacaftor/ivacaftor/tezacaftor, the first triple combination therapy for patients with the most common cystic fibrosis mutation.

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