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Rare Pediatric Disease

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March 19, 2024
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FDA approves Lenmeldy for early juvenile metachromatic leukodystrophy

FDA approves Lenmeldy for early juvenile metachromatic leukodystrophy

The FDA has approved Lenmeldy as the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy.

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February 21, 2024
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FDA grants rare pediatric disease designation to Duchenne muscular dystrophy drug

FDA grants rare pediatric disease designation to Duchenne muscular dystrophy drug

The FDA has granted rare pediatric disease designation to an investigational therapy for the treatment of Duchenne muscular dystrophy in those with mutations amenable to exon 44 skipping, according to the manufacturer.

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January 17, 2024
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FDA grants rare pediatric disease designation to alpha kinase inhibitor for ROSAH syndrome

FDA grants rare pediatric disease designation to alpha kinase inhibitor for ROSAH syndrome

The FDA has granted rare pediatric disease designation to an alpha-kinase 1 inhibitor to treat patients with retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache, or ROSAH, syndrome.

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December 15, 2023
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First patients dosed in clinical trial of Rett syndrome gene therapy

First patients dosed in clinical trial of Rett syndrome gene therapy

The first two female pediatric patients with Rett syndrome have been dosed in an ongoing phase 1/2 trial of investigational AAV9 gene therapy NGN-401, according to the manufacturer.

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October 03, 2023
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FDA removes partial clinical hold on IND for rare pediatric disease drug-device combo

FDA removes partial clinical hold on IND for rare pediatric disease drug-device combo

A Bay Area biotech company announced the FDA has lifted the partial clinical hold on EryDel SpA’s investigational new drug application for EryDex to treat ataxia-telangiectasia, a rare pediatric neurological disorder.

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August 09, 2023
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FDA grants rare pediatric disease designation to HG004 for inherited retinal diseases

FDA grants rare pediatric disease designation to HG004 for inherited retinal diseases

HG004, a gene replacement therapy, received rare pediatric disease designation from the FDA for the treatment of inherited retinal diseases caused by RPE65 mutations, according to a press release from HuidaGene.

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July 21, 2023
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FDA grants rare pediatric disease designation to neonatal lung therapy

FDA grants rare pediatric disease designation to neonatal lung therapy

The FDA has granted rare pediatric disease designation to vitamin A metabolic and reparative respiratory drugs manufactured by Advent Therapeutics to stop the development of bronchopulmonary dysplasia, according to a press release.

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March 01, 2023
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FDA approves first oral treatment for Friedreich’s ataxia

FDA approves first oral treatment for Friedreich’s ataxia

The FDA has approved Skyclarys, Reata Pharmaceuticals’ oral, once-daily medication to treat Friedreich’s ataxia in people aged 16 years and older.

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April 18, 2022
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Emerging therapeutics may improve outcomes in pediatric patients with rare disorders

Emerging therapeutics may improve outcomes in pediatric patients with rare disorders

SEATTLE — Clinicians should consider disease-modifying therapeutics when treating patients with rare pediatric disorders, according to an expert at the 2022 American Academy of Neurology annual meeting.

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January 04, 2022
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FDA grants rare pediatric disease designation to IMX-110 for rhabdomyosarcoma

FDA grants rare pediatric disease designation to IMX-110 for rhabdomyosarcoma

The FDA granted rare pediatric disease designation to IMX-110 for treatment of children with rhabdomyosarcoma.

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