RAIN
-
News
PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Patients May See Improved Outcomes with Ravulizumab
The findings showed that patients treated with ravulizumab demonstrated better adherence and persistence compared to those on other complement inhibitors. The ADVANTAGE study, a retrospective longitudinal analysis, included data from…
Read More » -
News
SICKLE CELL DISEASE | FDA Grants Orphan Drug Designation to AND017
Announced by KIND Pharmaceuticals on October 25, 2024, ODD is granted to a drug or biological product to prevent, diagnose, or treat a rare disease or condition that affects fewer…
Read More » -
News
Splenectomy as Treatment for Primary Warm Autoimmune Hemolytic Anemia (wAIHA)
Warm Autoimmune Hemolytic Anemia (wAIHA) occurs when the body’s immune system mistakenly produces antibodies that attack and destroy its own red blood cells. In this particular case, the patient presented…
Read More » -
News
APLASTIC ANEMIA | Hetrombopag Achieves Significant Hematological Responses in Newly Diagnosed Patients
In this study, newly diagnosed patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA) were initially treated with either a combination of cyclosporin A (CsA) and hetrombopag (HETROM+CsA group) or CsA alone…
Read More » -
News
REGULATORY APPROVAL | PiaSky Becomes the First Monthly Treatment for PNH in the EU
Paroxysmal Nocturnal Haemoglobinuria (PNH) is a rare and life-threatening blood condition where red blood cells are destroyed by the complement system – part of the innate immune system – causing…
Read More » -
News
JUST LISTEN | Voices of PK Deficiency Episode on the Impact of New International Standards
Host Dr. Rachael Grace is joined by Tamara Schryver of Thrive with Pyruvate Kinase Deficiency and Alejandra Watson from the PKD Foundation to shed light on these ground-breaking guidelines. Listeners…
Read More » -
News
NEW APPROVAL | FDA Endorses Piasky for Paroxysmal Nocturnal Hemoglobinuria
Piasky, developed by Roche, is a monoclonal antibody that targets complement protein C5. Crovalimab binds to C5 to prevent its cleavage, thus preventing the formation of the membrane attack complex…
Read More » -
News
CAD | Enjaymo Not Cost-Effective at Current Price, According to US Study
This conclusion was drawn from a cost-effectiveness study utilizing data from the Phase 3 CARDINAL clinical trial, which backed the treatment’s approval for CAD patients with a recent history of…
Read More » -
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…
Read More » -
News
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…
Read More »