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BioWorld - Friday, January 2, 2026
Home » Keywords » Phelan-McDermid syndrome

Items Tagged with 'Phelan-McDermid syndrome'

ARTICLES

Neurology/psychiatric

FDA grants orphan drug designation to BA-102 for Phelan-McDermid syndrome

April 22, 2025
Neuronos Ltd., a subsidiary of Beyond Air Inc., has announced BA-102 has been awarded U.S. orphan drug designation for the treatment of Phelan-McDermid syndrome.
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Girl in wheelchair

Neuren’s NNV-2591 positive in Angelman syndrome phase II trial

Aug. 13, 2024
By Tamra Sami
Neuren Pharmaceuticals Ltd.’s NNZ-2591 met the primary endpoints in a phase II trial in children with Angelman syndrome, with improvements seen in clinically important aspects of the disease, including communication, behavior, cognition and motor abilities, Neuren CEO Jon Pilcher said during an Aug. 9 conference call.
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Girl in wheelchair

Neuren’s NNV-2591 positive in Angelman syndrome phase II trial

Aug. 9, 2024
By Tamra Sami
Neuren Pharmaceuticals Ltd.’s NNZ-2591 met the primary endpoints in a phase II trial in children with Angelman syndrome, with improvements seen in clinically important aspects of the disease, including communication, behavior, cognition and motor abilities, Neuren CEO Jon Pilcher said during an Aug. 9 conference call.
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Brain clay model

Neuren’s NNZ-2591 meets phase II endpoints in Pitt Hopkins syndrome

June 4, 2024
By Tamra Sami
Neuren Pharmaceuticals Ltd.’s NNZ-2591 met the primary endpoints in a phase II trial in children with Pitt Hopkins syndrome, with improvements seen in communication, social interaction, cognition and motor abilities, according to top-line results.
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Brain clay model

Neuren’s NNZ-2591 meets phase II endpoints in Pitt Hopkins syndrome

May 29, 2024
By Tamra Sami
Neuren Pharmaceuticals Ltd.’s NNZ-2591 met the primary endpoints in a phase II trial in children with Pitt Hopkins syndrome, with improvements seen in communication, social interaction, cognition and motor abilities, according to top-line results.
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Brain clay model
Neurology/Psychiatric

FDA clears IND for Jaguar’s JAG-201 for genetic form of ASD and Phelan-McDermid syndrome

Feb. 1, 2024
Jaguar Gene Therapy LLC’s IND application for JAG-201, a gene therapy for a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS; 22q13.3 deletion syndrome), has been cleared by the FDA.
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Brain and DNA

Neuren’s NNZ-2591 meets endpoints in phase II trial in Phelan-McDermid syndrome

Dec. 19, 2023
By Tamra Sami
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
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Brain and DNA

Neuren’s NNZ-2591 meets endpoints in phase II trial in Phelan-McDermid syndrome

Dec. 18, 2023
By Tamra Sami
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
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Australia’s Neuren raises AU$20M to advance NNZ-2591 to the clinic in three orphan indications

June 30, 2020
By Tamra Sami
PERTH, Australia – Melbourne-based Neuren Pharmaceuticals Ltd. will be able to progress NNZ-2591 to the clinic for three orphan indications after raising AU$20 million (US$13.75 million) in a placement to institutional and sophisticated investors in Australia, New Zealand, Hong Kong and the U.K.
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Australia’s Neuren raises AU$20M to advance NNZ-2591 to the clinic in three orphan indications

June 29, 2020
By Tamra Sami
PERTH, Australia – Melbourne-based Neuren Pharmaceuticals Ltd. will be able to progress NNZ-2591 to the clinic for three orphan indications after raising AU$20 million (US$13.75 million) in a placement to institutional and sophisticated investors in Australia, New Zealand, Hong Kong and the U.K.
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