We thank Government of Andhra Pradesh for formulating a comprehensive rare disease policy

We thank Government of Andhra Pradesh for formulating a comprehensive policy to support patients suffering from Spinal Muscular Atrophy (SMA) and other rare diseases.

Spinal Muscular Atrophy (SMA) is a rare genetic disorder that severely affects muscle strength. Children with SMA often struggle to hold up their heads, swallow food, and even breathe properly.

For children diagnosed with SMA Type 1, a one-time Zolgensma gene therapy, costing around ₹16 crore, can significantly improve their condition if administered before the age of two. Families of children affected by SMA frequently turn to crowdfunding platforms to raise this enormous amount. For SMA Types 2, 3, and 4, several treatment options, including injectable and oral medications, are also available.

Many children in Andhra Pradesh are living with SMA. However, the State currently lacks a comprehensive system to:

*Identify children with this genetic disorder at an early stage.
*Maintain a registry of patients.
*Ensure timely diagnosis and treatment.
*Formulate a dedicated policy for SMA patients.

The case of a young girl named Punarvika, whose appeal to raise ₹16 crore for the Zolgensma injection went viral on social media, brought widespread attention to this issue. After nearly ₹10 crore had been raised through crowdfunding, Shri Nara Lokesh extended support to her family.

Recognizing both the challenges posed by SMA and the financial burden of crowdfunding, Shri Nara Lokesh announced in April 2026 that the Government of Andhra Pradesh would formulate a policy to ensure early identification, treatment, and comprehensive support for children affected by SMA in the State.

https://x.com/naralokesh/status/2045765724308980134

In October 2024, a grievance on this issue was submitted to Shri Nara Lokesh during the Praja Darbar program. The grievance was forwarded to the Director of Medical Education, Andhra Pradesh, for necessary action. However, the matter was closed without any concrete or meaningful resolution.

When a request was made in July 2026 to reconsider the grievance, Director of Medical Education, AP clarified that the Government of Andhra Pradesh is in the process of formulating a comprehensive policy for patients suffering from rare diseases. 

As part of this initiative, the Government proposes to establish:

*Awareness programs on rare diseases.
*A State Rare Disease Patient Registry.
*Free screening and diagnostic centers for early detection.
*Specialized training for doctors.
*Establishment of three referral centers.
*Creation of a Centre of Excellence for the treatment and management of rare diseases.

These are welcome and much-needed initiatives that can significantly improve the lives of children living with SMA and other rare diseases.

@PkGollamudi   Greviance and reply from DME : https://x.com/PkGollamudi/status/2079936319795376597