Rare Anaemias
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News
REGULATORY NEWS | EMA Accepts Rocket Pharma’s Gene Therapy Application for Fanconi Anemia
RP-L102 is a gene therapy containing autologous hematopoietic stem cells that have been modified to contain a functional copy of the FANCA gene. Mutations in FANCA hinder DNA repair and occur…
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EVENT | 1st RAIN Summit for Patients with Rare Anaemias | 18-19 May 2024
The workshop will present an excellent opportunity for patients to exchange knowledge on the latest scientific advances for Rare Anaemias, and share concerns and challenges. A unique occasion for a…
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REGULATORY NEWS | EU Backs Revolutionary Oral Monotherapy for Adult PNH Patients
The decision was hailed as a first step toward enabling patient access in European Union countries following a March 21 meeting of the Committee for Medicinal Products for Human Use…
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SPOTLIGHT | RAIN Endorses the First International Guidelines for Pyruvate Kinase Deficiency
Pyruvate kinase deficiency (PKD) is the most common cause of chronic congenital non-spherocytic haemolytic anaemia, impacting between one in 100,000 to one in 300,000 individuals globally. Those with PKD suffer…
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REGULATORY NEWS | AstraZeneca’s Voydeya Recommended for PNH Treatment in EU
Voydeya is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of care Ultomiris (ravulizumab) or Soliris (eculizumab) to address the needs of the approximately 10-20% of patients with PNH who…
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ELEVATING AWARENESS | 29 February Is Rare Disease Day
Rare Disease Day is the globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.…
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PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Ruxoprubart Scores FDA Orphan Drug Designation for PNH Treatment
FDA’s recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition. Ruxoprubart is currently undergoing a…
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APLASTIC ANEMIA | Incidence and Clinical Management in Spain Consistent with Other Studies
The study was carried out through a multicenter, ambispective, observational approach encompassing 7 Spanish hospitals, catering to roughly 3.91 million individuals, over the span from 2010 to 2019. The research…
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MYELODYSPLASTIC SYNDROMES | Advantages of Iron Chelation Revealed in Analysis of Low-Risk MDS
A recent groundbreaking study sheds new light on the effectiveness of iron chelation therapy (ICT) for patients suffering from low-risk myelodysplastic syndromes (LR-MDS). By delving into the impact of ICT…
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COLD AGGLUTININ DISEASE | ANX1502 Passes Phase 1 Trial, Proving Safe for Healthy Volunteers
Administered orally as a liquid suspension, ANX1502 demonstrated good tolerability, with no serious side effects reported. The drug’s pharmacokinetics also supported twice-daily dosing for a future proof-of-concept study. “We are…
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