SynaptixBio awarded £2m BioMedical Catalyst grant from Innovate UK
The grant will support clinical trials of rare disease therapy for leukodystrophy
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Tag: Rare diseases
Study reveals new insights into ‘gene misbehaviour’ in healthy populations
The findings could support investigations into human genetics and various complex diseases
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Researchers suggest treatments for rare diseases could reduce burden of kidney disease
Rare kidney diseases are a group of conditions which account for over 5% of people living with chronic kidney disease
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SynaptixBio says NHS diagnostic service for rare diseases could be expanded
A rare disease is defined as a condition that affects fewer than one in 2,000 people
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FDA approves ANDA of 20mg generic Nitisinone capsules
Nitisinone capsules are indicated for patients with hereditary tyrosinemia type 1 in combination with dietary restriction of tyrosine and phenylalanine
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Multi-million-pound funding for genomics research
Vital financing will help boost diagnosis, treatment and prospects of patient groups
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LifeArc and Great Ormond Street Hospital charity award £1.3m to tackle diseases
Funds will allow researchers to focus on improving the lives of children with rare conditions
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Neglected rare disease patients offered hope in new plan
Unmet need among over 7000 rare diseases is being addressed in a comprehensive action plan
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Sanofi’s oral BTK inhibitor rilzabrutinib fails Phase III trial
Rilzabrutinib missed primary and secondary endpoints as a treatment for pemphigus
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AZ scores final clearance for Alexion acquisition in the UK
UK approval of the acquisition comes just a week after the merger scored clearance in the EU
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EU green lights AZ, MSD’s rare disease therapy Koselugo
Drug approved to treat children with neurofibromatosis type 1 and plexiform neurofibromas
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NICE rejects rare blood cancer treatment Poteligeo in final appraisal
Final appraisal document said that clinical trial evidence for Poteligeo is ‘very uncertain’
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- Acesion Pharma launches phase 2 trial of AP31969 in atrial fibrillation
- Johnson & Johnson seeks EMA approval for teclistamab–daratumumab combination
- HUTCHMED begins phase 3 stage of pancreatic cancer trial
- Zenas reports strong phase 3 results for obexelimab in IgG4-related disease
- MRM Health collaborates with Oncode Institute and Netherlands Cancer Institute
