Two events ponder whether a large injection of cash could spark a breakthrough

Mark Mardell was left feeling 'humiliated' after he was told he could not board a flight.

UK's neurologist shortage continues to bite, says biggest PD audit

Background Multiple system atrophy (MSA) is a rare, rapidly progressive alpha-synucleinopathy characterized by autonomic dysfunction, parkinsonism and marked cerebellar ataxia, with very limited treatment options. N-acetyl-DL-leucine (ADLL) (Tanganil™), a racemic derivative of leucine is usually well tolerated and has shown promise in improving cerebellar symptoms and reducing REM sleep behavior disorder (RBD), a common non-motor dream-sleep disorder feature of alpha-synucleinopathy. Given this dual potential, we treated three patients with advanced-stage MSA under compassionate use rules. Case presentations Three patients with advanced-stage MSA and severe cerebellar symptoms—two of whom also had moderate RBD—were treated with ADLL (TanganilTM), titrated to 5g/day over 10 days. While both patients with RBD reported self-assessed decrease of RBD symptoms within 2–3 weeks on the full dosage (approximately four weeks after treatment initiation), all three patients had major worsening of gait and balance during the same period, leading to sudden falls and overall health decline. These adverse events prompted early discontinuation of therapy. Gait improved within two weeks after discontinuation of therapy in two patients. In parallel, the RBD phenotype reoccurred. In the third, who had very advanced MSA and concurrent infections, gait only slowly improved over time and it remains unclear whether his worsening of truncal ataxia was attributable to ADLL (TanganilTM), or whether it was related to his concurrent infections with slow recovery. Conclusions While ADLL improved RBD, it was poorly tolerated in these three patients with advanced-stage MSA of predominantly cerebellar type. Further studies are needed to evaluate its safety and efficacy in different, preferably early, stages of MSA.

Background DOPA decarboxylase (DDC) in cerebrospinal fluid (CSF) is an emerging Parkinson's disease (PD) biomarker, but its association with nonmotor symptoms is unclear. Objectives We aimed to de...

Parkinson's disease (PD) is a heterogeneous condition that presents variable clinical, neuropathological, and biomarker features. Disease progression can a...

The FDA plans to make a decision on Aug. 9 regarding a ketamine-based product that Pharmather hopes to investigate for Parkinson's dyskinesia.

Results from Phase IIb PADOVA and longer term follow-up data suggest clinical benefit on top of symptomatic treatment in early-stage Parkinson’s...

Very grateful to Gaby Rogers and the Channel 9 News team for highlighting the work of the Australian Parkinson’s Mission.

This webinar will cover the current medication pipeline, highlighting new therapies and their potential impact on symptom management.

When I was growing up, my mother had a long list of subjects unsuitable for polite conversation.

npj Parkinson's Disease - A personalised and comprehensive approach is required to suppress or replenish SNCA for Parkinson’s disease

Legal basis Art. 17 section 1 MAR - confidential information. Report content: In reference to current report 17/2024 regarding positive results of the Phase II clinical trial on a drug based on CPL'36...

Gothenburg, Sweden, March 5, 2025 - IRLAB Therapeutics AB (Nasdaq Stockholm: IRLAB A), a company that discovers and develops new treatments for Parkinson's disease, today announces topline results fro...