Archive · 19 Jun 2025
Parents Crowdfund for Rare Disease Gene Therapy
A baby girl has a rare disease called SMA Type I, which makes her muscles weak.
To help her, doctors gave her a special medicine that costs a lot of money.
Her parents asked people for money, and they raised enough to get the medicine.
The medicine helps by giving the baby the thing her body is missing.
If the medicine is given early, it can help the baby get better.
Doctors hope this medicine will help the girl grow stronger and healthier.
A 16-month-old girl with spinal muscular atrophy (SMA) Type I received a ₹16-crore gene therapy.
The child's parents raised ₹9 crore through crowdfunding to obtain the medicine.
The medicine, Onasemnogene abeparvovec, replicates a gene to help produce a crucial protein.
SMA Type I causes progressive muscle weakening, often leading to respiratory complications.
Doctors emphasize that early administration of the medicine leads to better outcomes.
- Who
- The 16-month-old girl and her parents.
- What
- A 16-month-old girl received gene therapy for spinal muscular atrophy (SMA) Type I.
- When
- The medicine was administered on Tuesday.
- Where
- At a city hospital in India.
- Why
- To treat spinal muscular atrophy (SMA) Type I, a rare genetic disorder that causes muscle weakness.
Challenges
Solutions
Affordability of Treatment
Challenges
High cost of treatment and reliance on crowdfunding creates a significant financial burden.
Solutions
While expensive, the treatment offers life-changing potential; funding through grants can help.
- Child's Age
- 16 months
- Disease
- Spinal Muscular Atrophy (SMA) Type I
- Medicine Cost
- ₹16 crore
- Funds Raised
- ₹9 crore
- Medicine Name
- Onasemnogene abeparvovec
- Mechanism
- Replicates SMN1 gene
- Symptoms
- Muscle weakness, respiratory complications