disease

Skyhawk Therapeutics Announces Positive First Interim Results in Patients from its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington’s Disease

SKY-0515 achieves dose-dependent reductions of mutant huntingtin (mHTT) protein, with 62% lowering at Day 84 on the 9mg daily oral…

1 week ago

Metabolon Partners with China Kadoorie Biobank to Advance Precision Health

The landmark multiomics study will examine how lifestyle, environmental, socioeconomic, and genetic factors play a significant role in shaping common…

2 weeks ago

Psoriatic Disease To Take Center Stage at the UN General Assembly: IFPA Calls for Greater Recognition of Psoriatic Disease During UN High-Level Meetings

STOCKHOLM, Sept. 11, 2025 /PRNewswire/ -- The International Federation of Psoriasis Associations (IFPA) spotlights psoriatic disease at the upcoming UNGA…

2 weeks ago

NanoNewron Awarded $2.5 Million in NIH STTR Phase 2 Grant to Advance its Innovative NN-840 Alzheimers Disease Program

The $2.5 Million in NIH STTR Phase 2 Grant allows NanoNewron to start the IND-enabling program for the NN-840 program…

2 weeks ago

Chiesi Global Rare Diseases Highlights Continued Commitment to Rare Disease Community at the International Congress of Inborn Errors of Metabolism (ICIEM) 2025

September 09, 2025 11:00 ET  | Source: Chiesi Global Rare Diseases Results presented focus on clinical insights on Fabry disease…

2 weeks ago

Junshi Biosciences Announces the Phase 3 Study of JS005 (IL-17A) for the Treatment of Moderate to Severe Plaque Psoriasis Met Primary Endpoints

SHANGHAI, Sept. 07, 2025 (GLOBE NEWSWIRE) -- Shanghai Junshi Biosciences Co., Ltd (Junshi Biosciences, HKEX: 1877; SSE: 688180), a leading…

3 weeks ago

U.S. FDA Approves Expanded Indication for VONVENDI [von Willebrand factor (Recombinant)] for Adults and Children with Von Willebrand Disease

− Approval Expands Use of VONVENDI to Include Routine Prophylaxis to Reduce the Frequency of Bleeding Episodes in Adults with…

3 weeks ago

London musician with brain tumour inspires Brighton triathlon fundraiser

Tuesday 26 August, 2025 A talented musician from London, who died from a rare brain tumour in January this year…

4 weeks ago

Abbott receives regulatory approval for the first denosumab biosimilar in Thailand, expanding access to bone disease treatment

Abbott's denosumab biosimilar is the first approved in Thailand, making advanced therapy for osteoporosis and cancer-related bone loss more affordable for…

1 month ago

A Life Rewritten: KFSHRC Treats Rare Disease After 22 Years of Specialized Care

RIYADH, Saudi Arabia, Aug. 24, 2025 (GLOBE NEWSWIRE) -- In a rare example of a lifelong bond between a patient…

1 month ago