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News about "Rare disease treatment"
EU Approves Chiesi's LOJUXTA for Pediatric HoFH Patients
The European Commission has approved Chiesi’s LOJUXTA (lomitapide) for children aged 5 years and older with Homozygous Familial Hypercholesterolaemia (HoFH), expanding treatment options for this ultra-rare genetic disorder.
Rare Disease Treatment | 25/06/2026 | By News Bureau
Chugai Pharma's Avastin Secures Approval in Japan for Additional Indication
Chugai Pharma’s Avastin has received approval in Japan for an additional indication, becoming the world’s first therapeutic option for neurofibromatosis type 2. The approval is supported by positive phase 2 clinical study results.
Rare Disease Treatment | 22/06/2026 | By News Bureau
FDA Grants Priority Review to Sanofi's Venglustat for Type 3 Gaucher Disease
Sanofi’s brain-penetrant investigational therapy venglustat has received FDA priority review after phase 3 data demonstrated positive efficacy and safety outcomes in patients with type 3 Gaucher disease.
Rare Disease Treatment | 29/05/2026 | By News Bureau
Pharming Secures EU Approval for Joenja as First Targeted Treatment for Rare Immune Disorder APDS
Pharming Group has received European Commission approval for Joenja, expanding treatment access for patients with activated PI3K delta syndrome and strengthening therapeutic options for rare immune disorders.
Rare Disease Treatment | 26/05/2026 | By News Bureau
Genethon and Ampersand Biomedicines Partner to Develop Precision Gene Therapy Vectors
Genethon and Ampersand Biomedicines join forces to engineer advanced AAV gene therapy vectors designed for greater tissue specificity, potentially enabling safer and more effective treatments for patients with rare genetic disorders.
Rare Disease Treatment | 25/05/2026 | By News Bureau | 103
Regenxbio Reports Positive Pivotal Trial Results for Duchenne Gene Therapy RGX-202
Regenxbio’s investigational gene therapy RGX-202 demonstrated strong microdystrophin expression, functional gains and a favourable safety profile in Duchenne muscular dystrophy patients, supporting plans for accelerated approval and a potential commercial launch in 2027.
Rare Disease Treatment | 15/05/2026 | By News Bureau
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