NiemannPick India

16/10/2025

🌿 Gratitude and Reflections-
Niemann-Pick Mini Symposium at AIIMS 🌿
🌿Niemann Pick awareness month.🌿

Today marked another meaningful milestone for the NiemannPick India as we came together at AIIMS, New Delhi, for the Niemann-Pick Mini Symposium, a day of learning, collaboration, and heartfelt connection.

Our deepest gratitude to AIIMS for providing this wonderful platform and for helping connect families, clinicians, and researchers. The journey with a rare disease is often lonely and difficult, and opportunities like this, where families can meet, share, and learn from one another, mean more than words can express.

We extend heartfelt thanks to all our distinguished speakers, delegates, and supporters who joined in person and virtually. Your time, insights, and encouragement mean the world to our small but determined rare disease community.

Special thanks to Dr. Pankaj Hari, Dr. Neerja Gupta, Dr. Madhulika Kabra, Dr. Ratna Puri, Dr. Sunita Bijarnia, Dr. B. S. Charan, Dr Seema Thakur, Dr. Mark Patterson, and Laura López for sharing their invaluable insights on Niemann-Pick Disease, helping families understand this complex condition a little better.

We are equally grateful to all the delegates and clinicians who took the time to engage with families, listen with empathy, and patiently address their questions. Your kindness and guidance brought reassurance and hope to many who often walk a challenging path.

At NiemannPick India, our journey continues, through advocacy, awareness, family support, and partnerships with clinicians, researchers, industry, and policymakers, to ensure no family walks this path alone.

💜 Together, we move forward with hope, courage, and gratitude.

aayog .india

13/10/2025

🌍 Why Niemann-Pick? Why Should You Care? What can you do?

A heartfelt thank you to our parents who have come forward to share the stories of their brave children — for education, awareness, and to inspire hope in others, despite their own ongoing challenges.

Most people have never heard of Niemann-Pick Disease (NPD) — a rare, life-limiting genetic disorder. But this post might just change your mind.

Niemann-Pick Type C (NPC) is one of the most devastating variants. It is an aggressive neurodegenerative condition that affects children and young adults. The toll it takes on families is unimaginable — emotionally, financially, and socially.

In India, there is no approved treatment for NPC.
Families face heartbreaking realities every day —
💔 How do you care for a child who has seizures every hour?
💔 How do you feed a child who can no longer swallow?
And this struggle continues for years, without access to the therapies that can slow the disease.

Globally, there are two FDA-approved drugs and several ongoing clinical trials that offer hope. Yet, in India, patients have no access to these medicines.

At NiemannPick India Charitable Trust, we are working tirelessly to change this.
We collaborate with the International Niemann-Pick Alliance, engage global patient groups, and partner with expert physicians across India to bring treatments and research access to our country.

Our upcoming Niemann-Pick Symposium on 16th October at AIIMS New Delhi is a step toward raising awareness and engaging India’s physicians, researchers, and policymakers to support this cause.

Comment below - you can meet us families and expert physicians at AIIMS. The program will also be available on YouTube.

Please amplify the voice of Rare Diseases — share, talk, and advocate.
Because timely diagnosis and access to treatment can save lives.

NiemannPick IndiaINPDA International Niemann-Pick Disease Alliance

Join us as we share the inspiring journey of a 3 young warriors living with Niemann-Pick Type C (NPC). Witness the challenges, courage, and hope that shape t...

08/10/2025

Join us for the Niemann-Pick Symposium 2025!

A platform bringing together clinicians, researchers, patient advocates, and families, all working toward better awareness, diagnosis, and treatment of Niemann-Pick Disease (ASMD & NPC) in India.

📅 Date: Thursday, 16th October 2025
🕙 Time: 10:00 AM – 1:00 PM (Followed by lunch)
📍 Venue: AIIMS, New Delhi (Mini Auditorium at Center for Dental Education And Research)

Don’t miss this opportunity to learn, connect, and be part of the movement for change.

👉 Register now:
https://docs.google.com/forms/d/1EPP-2bcsLcfq6oGrSILwvQpmDcxoORXl3KqeY9jfEps/edit

*D

06/10/2025

Niemann Pick Disease Mini Symposium –
16th October | AIIMS New Delhi 🧬

October marks the global Niemann Pick Disease Awareness Month, and we in India will have a special Symposium hosted by AIIMS, New Delhi – Centre of Excellence for Rare Diseases, in collaboration with Niemann Pick India Charitable Trust.

This event will bring together:
👩‍⚕️ Experts from expert clinicians, policy, and industry partners
👨‍👩‍👧‍👦 Patient families sharing their journeys and experiences
📚Sessions on understanding ASMD and NPC, supportive care, and preventive health approaches

💫 Join us to raise awareness, exchange ideas, and strengthen the rare disease ecosystem in India.

📅 Date: 16th October 2025
📍 Venue: AIIMS New Delhi (Hybrid mode)
✉️ For registration and participation: please email niemannpickindia@gmail.com

Together, let’s drive awareness, inclusion, and access for the Niemann Pick community.



NiemannPick India INPDA International Niemann-Pick Disease Alliance NITI Aayog AIIMS, New Delhi, India Ministry of Health and Family Welfare, Government of India Sanofi India

02/10/2025

🔬 New Research Alert for ASMD (NirmannPick 😎

We’re excited to share this recent publication in the Orphanet Journal of Rare Diseases that highlights important developments in the understanding of ASMD

📖 Read here: https://ojrd.biomedcentral.com/articles/10.1186/s13023-025-03997-6

At NiemannPick India, our mission is to bring patients, caregivers, clinicians, and researchers together to improve awareness, access, and research for NPD in India.

We believe research like this is a crucial step towards:
✅ Better understanding the disease
✅ Accelerating diagnosis
✅ Expanding access to care
✅ Building global collaborations

💡 We invite experts, researchers, and policymakers in India to connect with us and explore how such global insights can be translated into action for Indian patients.

Together, we can ensure that Niemann-Pick patients in India are not left behind.

Background Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder caused by SMPD1 mutations, resulting in sphingomyelin accumulation and diverse manifestations. Olipudase alfa, an enzyme replacement therapy, has shown efficacy in treating non-neurological symptoms of ASMD, whil...

30/09/2025

Story of Little Sarvika Sri

A routine check-up showed her liver was enlarged. Soon, her growth started to delay.

Tests confirmed Niemann–Pick Disease Type B. Doctors say costly ERT can save her, but treatment hasn’t started despite government registration and endless appeals.

Her parents are watching their child suffer while policies delay doesn't help.

Please share Sarvika’s story, raise your voice, and help us reach the right support.

Together, we can give her a chance at life.

25/09/2025

The Hidden Struggle of Rare Disease Diagnosis.

For families facing a rare disease, the hardest part often isn’t treatment, it’s getting a diagnosis.
Many spend years searching for answers, moving from doctor to doctor, test to test, while their loved one’s health hangs in the balance.

The challenges are real:
- Symptoms that look “ordinary” but hide something rare.
- Limited awareness among healthcare providers.
- Specialized tests that are hard to access.
- The long wait for genetic results.

Behind every delay is a child, a parent, a family waiting for hope.

By raising awareness, pushing for better testing, and supporting research, we can shorten the journey, so families get answers sooner, and care when it matters most.

Because no family should have to fight in the dark.

25/09/2025

This is a ASMD perspective index. A wonderful tool built for NiemannPick community by

22/09/2025

The INPDA International Niemann-Pick Disease Alliance face-to-face meeting brought together every voice that matters in the Niemann-Pick journey.

🔬 Researchers shared their cases and insights, deepening our understanding of NPD.
💊 Industry partners presented their perspectives on current and future therapies.
📊 International Niemann-Pick Disease Registry (INPDR) the global registry, showed how data can drive faster and stronger research outcomes.
👩‍⚕️ Physicians offered their expertise and perspectives on caring for patients.
🌐 Global Patient Associations shared lived experiences, advocacy efforts, and the fight for access to medicines.

For 5 days, we worked side by side — identifying gaps, building solutions, and strengthening the bridges between science, medicine, and families.

There is so much work in progress, so much determination to understand this disease better. And through it all, one thing is clear: hope raises when we stand together.

18/09/2025

🤝✨ Breaking the ice at Iguazú Falls as we meet, connect, and understand each other better.
The spirit of collaboration and friendship is already in the air as we get ready for the INPDA Conference beginning tomorrow! 💙

17/09/2025

🚫 Myth: Many people think that Niemann-Pick Disease only affects children. This is because symptoms often begin early in life, like developmental delays, difficulty walking, or enlarged organs in young kids.

✅ Fact: While symptoms often begin in early childhood, Niemann-Pick doesn’t only impact kids. Teenagers and even adults can be diagnosed too. And the way symptoms show up can look very different at every age.

Niemann-Pick doesn’t have a one-size-fits-all journey.
This rare genetic condition affects people across all stages of life. That’s why awareness, early diagnosis, and research are so important.

Did you know this before? Share in the comments.