Neurology

ProQR expands partnership with RSRT to advance Rett Syndrome therapy

ProQR Therapeutics has expanded its partnership with the Rett Syndrome Research Trust (RSRT), receiving an additional $8.1m in funding to support the advancement of Rett Syndrome therapy, AX-2402 into clinical trials.

FDA grants ODD to Ractigen Therapeutics’ RAG-21 for ALS treatment

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Ractigen Therapeutics' new therapy, RAG-21, targeting the fused in sarcoma (FUS) gene, for amyotrophic lateral sclerosis (ALS) treatment.

Asha Therapeutics secures grant for ALS therapeutic development

Asha Therapeutics has received a grant from the ALS Association through its Lawrence and Isabel Barnett Drug Development Program for advancing ASHA-624 to treat amyotrophic lateral sclerosis (ALS).

FDA issues CRL to PharmaTher’s ketamine ANDA

The US Food and Drug Administration (FDA) has issued a complete response letter (CRL) to PharmaTher concerning its ketamine abbreviated new drug application (ANDA).

FDA grants orphan drug status to Papillon Therapeutics’ PPL-001

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Papillon Therapeutics’ PPL-001, aimed at treating Friedreich's ataxia, a rare neurodegenerative disorder.

FDA grants orphan drug status for RedHill’s opaganib for neuroblastoma

The US Food and Drug Administration (FDA) has granted orphan drug designation to RedHill Biopharma's opaganib for the treatment of neuroblastoma, a childhood cancer.

FDA approves Alpha Cognition’s ZUNVEYL for Alzheimer’s treatment

The US Food and Drug Administration (FDA) has approved Alpha Cognition's oral therapy, ZUNVEYL, for the treatment of mild-to-moderate Alzheimer's disease.

Leqembi gains approval for Alzheimer’s treatment in Israel

BioArctic's partner Eisai has announced that Leqembi, has received approval in Israel for the treatment of Alzheimer's disease (AD), specifically for patients with mild cognitive impairment or mild dementia, the same stages targeted in clinical trials.

Augustine raises €17m to advance lead candidate into trials for neuro conditions

Augustine Therapeutics has secured €17m ($18.5m) in a Series A funding round's initial close for advancing its lead candidate, AGT100216, into a Phase I/II first-in-human clinical trial next year to treat neurological conditions.