Daily Current Affairs : 21-October-2023
Niemann-Pick disease, a rare genetic disorder impacting fat metabolism, has become a focal point for parents in India. These parents are passionately urging the Indian government to include this condition in the National Policy for Rare Diseases, aiming to raise awareness and provide crucial support for affected families.
Understanding Niemann-Pick Disease
Niemann-Pick disease comprises a group of inherited metabolic disorders where abnormal lipid accumulation occurs in vital organs like the brain, spleen, liver, lungs, and bone marrow. Its manifestations include an enlarged liver and spleen, difficulty in movement coordination, frequent respiratory infections, and slurred speech, among other symptoms. This condition follows an autosomal recessive pattern, necessitating both gene copies to be faulty for the disease to surface.
Prevalence and Challenges
Niemann-Pick disease is a rare condition, affecting only one in 250,000 individuals. Despite its rarity, it poses significant challenges due to its expensive treatment requirements, making it financially burdensome for affected families to manage the disease effectively.
National Policy for Rare Diseases: A Beacon of Hope
The National Policy for Rare Diseases stands as a beacon of hope for individuals grappling with uncommon medical conditions. This government initiative aims to offer comprehensive support to those afflicted, ensuring they receive the necessary care and financial assistance.
The Significance of Inclusion
The inclusion of Niemann-Pick disease in the National Policy for Rare Diseases holds immense significance:
- Financial Support: Inclusion in the policy would mean eligible patients receive crucial financial aid, easing the burden on families already coping with substantial medical expenses.
- Access to Life-saving Therapy: One pivotal aspect of this inclusion is providing access to Xenpozyme, an expensive enzyme replacement therapy. Recently, the Drug Controller General of India allowed a pharmaceutical company to import and market Xenpozyme following approval from the US FDA. This breakthrough therapy can significantly enhance the quality of life for those afflicted by Niemann-Pick disease, making the inclusion in the policy even more critical.
Important Points:
- Niemann-Pick Disease Overview:
- Inherited metabolic disorder causing abnormal lipid buildup in vital organs.
- Symptoms include enlarged liver and spleen, movement coordination issues, respiratory infections, and slurred speech.
- Occurs in an autosomal recessive pattern, requiring both faulty gene copies for disease manifestation.
- Prevalence and Challenges:
- Rare disease affecting 1 in 250,000 individuals.
- Expensive treatment poses significant financial burden on affected families.
- National Policy for Rare Diseases:
- Government initiative providing comprehensive support for rare medical conditions.
- Aims to offer financial assistance and essential care to affected individuals.
- Significance of Inclusion:
- Financial Support:
- Inclusion in the policy provides crucial financial aid to affected families.
- Access to Xenpozyme Therapy:
- Inclusion grants access to Xenpozyme, an expensive enzyme replacement therapy.
- Recently approved by the US FDA and permitted for import and marketing in India.
- Xenpozyme significantly enhances the quality of life for Niemann-Pick disease patients.
- Financial Support:
- Parental Advocacy:
- Parents are passionately urging the Indian government to include Niemann-Pick disease in the National Policy for Rare Diseases.
- Advocacy focuses on raising awareness and garnering crucial support for affected families.
Why In News
Parents of children grappling with Niemann-Pick disease, a rare genetic disorder that disrupts fat metabolism, are passionately urging the Indian government to recognize their plight and incorporate the disease into the National Policy for Rare Diseases, advocating for better research, funding, and support for affected families.
MCQs about Niemann-Pick Disease
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What is the primary aim of the National Policy for Rare Diseases in India?
A. To raise awareness about Niemann-Pick disease.
B. To provide financial support and essential care for individuals with rare medical conditions.
C. To import and market advanced therapies like Xenpozyme.
D. To solely focus on reducing the prevalence of Niemann-Pick disease.
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How does Niemann-Pick disease affect individuals and their families?
A. It causes hair loss and skin rashes.
B. It leads to difficulty in movement coordination and respiratory infections, posing a significant financial burden on families due to expensive treatments.
C. It primarily affects vision and hearing.
D. It results in mild fatigue and occasional headaches.
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What is Xenpozyme?
A. A rare disease awareness campaign in India.
B. An enzyme replacement therapy for Niemann-Pick disease.
C. A government initiative to address rare diseases.
D. A research organization dedicated to genetic disorders.
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Why are parents advocating for the inclusion of Niemann-Pick disease in the National Policy for Rare Diseases?
A. To gain access to free medical consultations.
B. To raise funds for research on the disease.
C. To ease the financial burden by receiving financial support and granting access to therapies like Xenpozyme.
D. To lobby for the construction of specialized medical facilities.
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