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Sickle Cell
Disease
Sickle Cell
Disease Rare
Disease
01
Introduction Epidemiology
02
Pathogenesis
03
Symptoms
04
Treatment
05 06
AboutCompanies &
Drugs
Introduction
Sickle cell disease (SCD) is an inherited red
blood cell disorder caused by a structural
abnormality of hemoglobin called sickle
hemoglobin (HbS) which encodes hemoglobin
subunit β. HbS is inherited in an autosomal
recessive way and can occur due to
homozygosity for HbS (HbSS).
Sickle cell disease (SCD) is an inherited red blood cell disorder caused by a
structural abnormality of hemoglobin called sickle hemoglobin (HbS) which
encodes hemoglobin subunit β. HbS is inherited in an autosomal recessive way and
can occur due to homozygosity for HbS (HbSS).
HbS is functional and soluble when oxygenated, but upon deoxygenation it
polymerizes, leading to the generation of deformed red blood cells known as
sickled cells and dense erythrocytes. Due to increased global and intra-EU mobility
and migration, the number of patients with SCD in Europe has increased.
HbS is functional and soluble when oxygenated, but upon deoxygenation it
polymerizes, leading to the generation of deformed red blood cells known as
sickled cells and dense erythrocytes. Due to increased global and intra-EU mobility
and migration, the number of patients with SCD in Europe has increased. According
What is Sickle Cell Disease?
Epidemiology
● It is estimated that SCD affects approximately 100,000 Americans.
● It occurs among about 1 out of every 365 Black or African-American births,
about 1 out of every 16,300 Hispanic-American births, and about 1 in 13 Black
or African-American babies is born with sickle cell trait.
● the global meta-estimate for the birth prevalence of homozygous sickle cell
disease was 111.91 per 100 ,000 live births and that for heterozygous sickle
cell disease was 4229.72 per 100 ,000, and it was least in Europe, i.e., 803.57
per 100,000 live births.
● That demographical projections estimated that the annual number of newborn
babies with SCA worldwide will exceed 400,000 by 2050.
Sickle Cell Disease Pathogenesis
The momentous pathophysiological occasion in the erythrocytes of
people with the different sickle conditions is the intracellular
conglomeration or polymerization of sickle hemoglobin (HbS).
The degree of polymerization is dictated by intracellular hemoglobin
piece (% HbS and % HbS A, A2, and F), focus (MCHC and % of thick
cells), and oxygen immersion, just as minor factors like intracellular pH
and DPG fixation
Sickle Cell Disease Symptoms
1. Anemia
2. Scenes of Pain
3. Expanding of hands and feet
4. Incessant diseases
5. Deferred Growth or Puberty
6. Vision Problems
• The treatment starts with the recognition of pain in the sickle cell
disease patients. Recurrent episodes of acute, severe pain are the
hallmark of SCD.
• The pain is highly variable both within and among patients, and is
the result of complex and poorly understood interactions between
biological and psychosocial factors.
Sickle Cell Disease Treatment
Sickle Cell Disease Companies & Drugs
• Bristol-Myers Squibb
• Emmaus Life Sciences, Inc
• Pfizer Inc.
• Micelle BioPharma Inc.
• AstraZeneca
• BlueBird Bio
• Agios Pharmaceuticals
• Droxia
• Endari
• Rivipansel
• SC411 (Altemia)
• Ticagrelor
• LentiGlobin BB305
• AG-348
Companies Drugs
Thank You

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Sickle Cell Disease Market Growth factors and Market Forecast 2030

  • 4. Introduction Sickle cell disease (SCD) is an inherited red blood cell disorder caused by a structural abnormality of hemoglobin called sickle hemoglobin (HbS) which encodes hemoglobin subunit β. HbS is inherited in an autosomal recessive way and can occur due to homozygosity for HbS (HbSS).
  • 5. Sickle cell disease (SCD) is an inherited red blood cell disorder caused by a structural abnormality of hemoglobin called sickle hemoglobin (HbS) which encodes hemoglobin subunit β. HbS is inherited in an autosomal recessive way and can occur due to homozygosity for HbS (HbSS). HbS is functional and soluble when oxygenated, but upon deoxygenation it polymerizes, leading to the generation of deformed red blood cells known as sickled cells and dense erythrocytes. Due to increased global and intra-EU mobility and migration, the number of patients with SCD in Europe has increased. HbS is functional and soluble when oxygenated, but upon deoxygenation it polymerizes, leading to the generation of deformed red blood cells known as sickled cells and dense erythrocytes. Due to increased global and intra-EU mobility and migration, the number of patients with SCD in Europe has increased. According What is Sickle Cell Disease?
  • 7. ● It is estimated that SCD affects approximately 100,000 Americans. ● It occurs among about 1 out of every 365 Black or African-American births, about 1 out of every 16,300 Hispanic-American births, and about 1 in 13 Black or African-American babies is born with sickle cell trait. ● the global meta-estimate for the birth prevalence of homozygous sickle cell disease was 111.91 per 100 ,000 live births and that for heterozygous sickle cell disease was 4229.72 per 100 ,000, and it was least in Europe, i.e., 803.57 per 100,000 live births. ● That demographical projections estimated that the annual number of newborn babies with SCA worldwide will exceed 400,000 by 2050.
  • 8. Sickle Cell Disease Pathogenesis The momentous pathophysiological occasion in the erythrocytes of people with the different sickle conditions is the intracellular conglomeration or polymerization of sickle hemoglobin (HbS). The degree of polymerization is dictated by intracellular hemoglobin piece (% HbS and % HbS A, A2, and F), focus (MCHC and % of thick cells), and oxygen immersion, just as minor factors like intracellular pH and DPG fixation
  • 9. Sickle Cell Disease Symptoms 1. Anemia 2. Scenes of Pain 3. Expanding of hands and feet 4. Incessant diseases 5. Deferred Growth or Puberty 6. Vision Problems
  • 10. • The treatment starts with the recognition of pain in the sickle cell disease patients. Recurrent episodes of acute, severe pain are the hallmark of SCD. • The pain is highly variable both within and among patients, and is the result of complex and poorly understood interactions between biological and psychosocial factors. Sickle Cell Disease Treatment
  • 11. Sickle Cell Disease Companies & Drugs • Bristol-Myers Squibb • Emmaus Life Sciences, Inc • Pfizer Inc. • Micelle BioPharma Inc. • AstraZeneca • BlueBird Bio • Agios Pharmaceuticals • Droxia • Endari • Rivipansel • SC411 (Altemia) • Ticagrelor • LentiGlobin BB305 • AG-348 Companies Drugs