Thalassemia org

Thalassemia org thalassemia.org Thalassemia (also spelled thalassaemia) is an inherited autosomal recessive blood disease that originated in the Mediterranean region.

In thalassemia the genetic defect, which could be either mutation or deletion, results in reduced rate of synthesis or no synthesis of one of the globin chains that make up hemoglobin. This can cause the formation of abnormal hemoglobin molecules, thus causing anemia, the characteristic presenting symptom of the thalassemias. Thalassemia is a quantitative problem of too few globins synthesized, whereas sickle-cell disease (a hemoglobinopathy) is a qualitative problem of synthesis of an incorrectly functioning globin. Thalassemias usually result in underproduction of normal globin proteins, often through mutations in regulatory genes. Hemoglobinopathies imply structural abnormalities in the globin proteins themselves. The two conditions may overlap, however, since some conditions that cause abnormalities in globin proteins (hemoglobinopathy) also affect their production (thalassemia). Thus, some thalassemias are hemoglobinopathies, but most are not. Either or both of these conditions may cause anemia. The two major forms of the disease, alpha- and beta- (see below), are prevalent in discrete geographical clusters around the world - it is presumed associated with malarial endemicity in ancient times. Alpha is prevalent in peoples of Western African and South Asian descent. It is nowadays found in populations living in Africa and in the Americas. It is also found in Tharu in the Terai region of Nepal and India. and is believed to account for much lower incidence of morbidity and mortality, accounting for the historic ability of Tharus to survive in heavily malarial areas where others could not. Source
Description above from the Wikipedia article Thalassemia, licensed under CC-BY-SA full list of contributors here. Community Pages are not affiliated with, or endorsed by, anyone associated with the topic.

Great News -
25/12/2025

Great News -

Agios upgraded after FDA approval of Aqvesme for thalassemia. Explore AGIO’s financial outlook, upside potential, and future catalysts. Click here to read.

26/11/2025

- India has marked an important milestone in genomic medicine with the launch of BIRSA 101, the country’s first indigenous CRISPR-based for sickle cell disease (SCD).
Developed by the CSIR Institute of Genomics and Integrative Biology (IGIB), the therapy uses technology to edit a patient’s own stem cells, enabling the production of healthy red blood cells and addressing the genetic cause of the disorder.
remains especially prevalent among tribal communities in central and eastern India, and BIRSA 101—named in honour of tribal independence activist Birsa Munda—is specifically designed with this public health need in mind. Early clinical work included three patients supported by the Department of Science & Technology and the Ministry of Tribal Affairs, Government of India.
In parallel, CSIR-IGIB has partnered with the Serum Institute Of India to transfer the underlying gene-editing technology and prepare for scaled-up manufacturing, as well as Phase II and III .
For further details, visit: https://www.digitalhealthnews.com/india-launches-first...

https://www.businesswire.com/news/home/20210921005978/en/bluebird-bio-Submits-Biologics-License-Application-BLA-to-FDA-f...
22/09/2021

https://www.businesswire.com/news/home/20210921005978/en/bluebird-bio-Submits-Biologics-License-Application-BLA-to-FDA-for-betibeglogene-autotemcel-beti-cel-Gene-Therapy-for-Patients-With-%CE%B2-thalassemia-Who-Require-Regular-Red-Blood-Cell-Transfusions?fbclid=IwAR2uesmCl4zpf1TTOMREAcnwt6RQlWefm-6xUunM4_1xluKv7Z4AmP7upQg #.YUtl9dyDwNE.facebook

bluebird bio, Inc. (Nasdaq: BLUE) today announced it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food

Meet the person who is brains behind our Initiative who is a Thalassemia Warrior himself. Founder of Team PAPT, Anshul...
30/04/2020


Meet the person who is brains behind our Initiative who is a Thalassemia Warrior himself. Founder of Team PAPT, Anshul Goel.
---“Born on 29th December 1999, it was not more than five months when I suffered high fever and was later detected with a genetic blood disorder called Thalassemia.I suffer from the type Beta Thalassemia Major which is one of the most severe forms of the disease.So since the age of five months till now I have been regularly taking routine blood transfusions along with several other oral medications.Now I need to take routine blood transfusions every 20-22 days in order to sustain life.Due to the frequent blood transfusions, the veins in my hands are repeatedly punctured which damages them.It is often difficult to insert an IV which sometimes causes unbearable pain due to repeated pricking. I realised it much earlier in life that pain is something which I will have to endure every now and then and soon came to terms with it. My parents have provided me with the best treatments available for managing the disorder, our hopes being with the gene therapy which is currently undergoing trials at various locations abroad.
It was on 12th August 2011 when the world came crashing down on me again. I met with an accident when I had gone to the hospital to undergo my routine blood transfusion. I slipped on the stairs which weren’t mopped properly after a torrential downpour. Later we realised my femur bone broke into two and I had to be operated to set it back. Five rods were inserted in my leg and in the span of about 6 months post the operation, I learned to walk again after intensive extremely painful physiotherapy.Also during this phase I got detected with Hepatitis C. It came into me through untreated blood transfusion which was only detected during my pre surgery blood tests. The treatment for the same was very expensive and exhaustive but I came out of it too. Later, I started with an awareness program on Thalassemia and formed a team – Team PAPT (Project Awareness and Prevention of Thalassemia)as I don't want any more people to face the hardships that I had/have to face because of Thalassemia.I survived through many things and what motivates me to live is to work for the cause I shared in the last few lines. I want to make people aware about this. That's my aim.”


Thalassemics India Thalassemics Gujarat Thalassemia Society Thalassemia Prevention thalassemia Thalassemia org

30/04/2020

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