NiemannPick India

09/04/2026

This is not just a story from our community — it is one that feels deeply personal.

Guncha and her family are like extended family to many of us. We have witnessed their journey closely — a journey of strength, love, and unimaginable courage.

Many of us remember Aizha — her innocence, her beautiful smile, and the quiet bravery with which she faced Niemann-Pick Disease Type C (NPC).

There are no easy answers to why our children go through such pain. But what this story reminds us is something only this community truly understands — the complex, heartbreaking moment when a parent feels relief that their child is no longer suffering.

Guncha’s courage to share her journey continues to give strength to many families who feel alone. Stories like these remind us that we are not alone — we stand together.

We thank Dr Sarath R S for sensitively capturing and bringing forward this story for the world to understand. Stories like these help bridge the gap between science and humanity.

Wishing strength to all rare disease warriors and their families.
And hoping, with all our collective effort, that the world becomes kinder — one story, one step at a time.

NiemannPick India INPDA International Niemann-Pick Disease Alliance

Aizah’s NPC journey reveals the hidden reality of rare disease: where the weight of societal stigma and judgment is often as heavy as the genetic battle itself.

08/03/2026

Happy Women’s Day to the incredible mothers of rare disease children.

Many mothers in the rare disease community lovingly put their careers on hold to become full-time caregivers for their children.
They become doctors, nurses, therapists, teachers, and advocates — all driven by unconditional love.

Despite the challenges, these mothers continue their journey with strength, patience, and hope, finding joy in every small victory their children achieve.

But we also dream of a future where mothers do not have to sacrifice their careers.

With early diagnosis, timely treatment, and better access to therapies, children with rare diseases can live fuller lives — going to school, playing, and growing like other children. And mothers can return to their professional journeys with confidence.

Today we celebrate the resilience, love, and courage of Rare Mothers everywhere.

💜 You are the heart of every rare warrior’s journey.

Happy Women’s Day.

03/03/2026

NiemannPick India had a busy year end 2025. Thank you INPDA International Niemann-Pick Disease Alliance for covering the work we do in India.
Together for NPD

When we shared the latest INPDA Newsletter on Rare Disease Day, one important update was unintentionally missed — and we don't want our community to miss it.

We’re pleased to now share the update from NiemannPick India Charitable Trust, highlighting their ongoing work and initiatives supporting the Niemann-Pick community in India.

The update has now been added to the INPDA website, and we invite our community to take a moment to read it and learn more about the work being carried out.

Read the update on the INPDA website: https://www.inpda.org/niemannpick-india/

Thank you to Niemann-Pick India for their continued dedication to the community!

28/02/2026

Rare diseases need more voices and stronger advocacy—so patients receive timely diagnosis and equitable access to treatment they deserve.

On this Rare Disease Day, we are grateful to VOICE OF HEALTHCARE for featuring the work of NiemannPick India. Every voice raised and every story covered creates another chance—for awareness, for action, and for hope.

A big shoutout to all the voices featured—your tireless, often unseen contributions are shaping the rare disease landscape in India.

India’s rare disease landscape is being transformed by a community of extraordinary individuals whose commitment goes far beyond their professions. In this spec

01/01/2026

A New Year. A New Dose of Hope.

As the clock was turning to 2026, something truly extraordinary happened.
Yesterday, a child with NPC took her very first dose of a clinical trial medication by Azafaroz — right here in India.

A small pill.
A brave child.
And a giant leap of hope for countless rare disease families.

This isn’t just a medical milestone — it’s a message the New Year delivered:
Hope is arriving. Science is listening. And our children matter.

For the first time ever, an NPC clinical trial is being conducted in India, and this moment belongs to every family that kept believing when the road felt endless.

As you watch this little warrior take that pill, remember — sometimes the most powerful New Year resolutions come wrapped in courage, resilience, and a whole lot of love.

With gratitude, NPD families in India thank AZAFAROS B.V. And Sanofi for your unwavering support of Niemann-Pick warriors in India

Here’s to 2026 — where our work continues on -

🌿 more clinical trial sites,
💎rightful ERT access under NPRD 2021,
🍏uninterrupted care under NPRD 2021 for life long treatment and support

Because this year didn’t just say Happy New Year… It said, “Let’s begin.” 💫

Wishing everyone a very hopeful, healthy, and happier 2026! 🌟💙

from NiemannPick families in India NiemannPick India

18/12/2025

Some journeys shape a lifetime.

Aizah was a precious dream, here for a short while, yet forever changing lives.

It was her parents, spending time with rare disease families in Delhi, who found a deeper purpose through her — to stand beside children who fight silently and families who hold onto hope.

On her birthday, we remember Aizah by choosing compassion and speaking up for little lives like hers.

Aizah is now at peace, but she remains forever in the hearts of NiemannPick families.
Our promise remains — every warrior deserves access to medicine.

08/12/2025

Has the Indian government failed families living with Rare Diseases?

Rupsa Chakraborty testimony in this video lays bare the pain, uncertainty, and daily battles that thousands of families face across India. Thank you, Rupsa, for speaking with courage and for standing with a community that is so often overlooked.

It is heartbreaking that Group 3 Rare Disease patients—those requiring lifelong, high-cost treatments—receive only a one-time allocation of ₹50 lakh. The very clause meant to support “lifelong” therapy is fundamentally flawed. Many children start treatment, only to have it stopped midway because the funds are exhausted. And when families turn to the courts, the response is: wait until March 2026.
Our only prayer is that these children survive long enough to hear that judgment.

ASMD (Niemann-Pick Type A/B) is a recognised Rare Disease with a proven enzyme replacement therapy. Yet, because it has not been included under the NRDP 2021, only handful patients have access through pharma’s humanitarian program; otherwise not a single patient in India has access to this life-saving treatment through the Rare Disease Policy. Each passing month risks irreversible damage. Some families may lose their children while policies remain frozen.

We continue to advocate—relentlessly, tirelessly—because parents cannot afford to give up. The fight for timely, equitable access to treatment must continue until every child with a Rare Disease in India receives the care they deserve.

Watch Rupsa Chakraborty bring this out fearlessly backed with Data

Speaker: Rupsa Chakraborty, 2023 NPF Rare Disease Fellow; MSc Global Health Policy, LondonSchool of EconomicsThis training is sponsored by Fondation Ipsen.Th...

02/12/2025

A platform that truly brings together scientists, clinicians, industry leaders, students, startups, policymakers, and patient support groups — that is REDRESS2025 by Tata Institute for Genetics and Society, India and Organization for Rare Diseases India

On behalf of NiemannPick India, we are grateful for the opportunity provided to present the patient voices of NPD to this prestigious forum.

For years, we have heard comments like, “Why involve patient? What will patients know about science, genomics, or gene therapy? All they care about is treatment…”
That narrative is changing — and TIGS is change that.

By placing patients at the centre of scientific conversations and ensuring we are part of the decision-making table, TIGS is redefining how rare disease solutions are shaped in India.

The two-day conference was much more than talks and presentations — it was two days of hope for a brighter future for rare diseases.

For us at NiemannPick India, it meant:

🔹 Networking with experts
🔹 Seeking guidance and collaboration
🔹 Strengthening our advocacy for Niemann-Pick
🔹 Learning from the best minds in the rare disease ecosystem
🔹 Building bridges for research, diagnosis, and clinical readiness

We return with renewed energy — sleeves rolled up, heads down — ready to act on every takeaway and continue pushing forward for our children, families, and the entire rare disease community.

Organization For Rare Diseases India Institute of Genetics and Society

26/11/2025

I just read this deeply troubling piece by Global Health NOW on India’s “funding failure” for rare genetic disease care — and it hits far too close to home.

To our Government / Policymakers: This is more than a policy issue — it’s about real families, real children.

Rare Disease families often feel like startup founders, scrambling to raise capital for something existential. The difference? These “founders” are parents, and the product is their child’s life.

Have you ever paused to think what the conversations are like around the dining table in our homes? It’s never about normal childhood stuff. Instead:
• “How do we raise the next installment for treatment?”
• “Can we find a donor?”
• “Will the government release the money next month?”

And our children—they hear it too. The anxiety, the fear, the uncertainty become part of childhood. It’s a scar that never quite heals.

The NPRD 2021 cap of ₹50 lakh is far below what many life-saving therapies require. Only about 30% of applications get approved, and even then, funding stops once the ceiling is reached. CoEs are under-utilizing their budgets, and despite a court-mandated ₹974-crore National Rare Diseases Fund, families are still left waiting — and suffering.

We are not asking for charity — we are asking for justice, dignity, and a policy that actually works.
We need:
• A permanent, adequately funded national rare disease fund
• Flexibility beyond the ₹50 lakh cap for lifelong, high-cost therapies
• Accountability and full utilization of CoE budgets
• Transparent, predictable funding processes
• Integration of rare disease care into mainstream health planning

For too long, rare disease has remained a whisper. It’s time we make it impossible to ignore.

And hoping that the conversation around the dinning table will be more fun!!

India’s rare disease policy was hailed in 2021 as a turning point for patients, but funding problems have meant that some wait for medicines that never arrive.

22/11/2025

We are delighted to share that Niemann Pick India Charitable Trust, in partnership with the Ministry of Health — Institute of Health & Family Welfare, Government of West Bengal, successfully organized a One-Day Workshop on Niemann Pick Disease and other Lysosomal Storage Disorders at Swasthya Bhawan, Kolkata.

This workshop brought together healthcare professionals, policymakers, patient groups and industry partners to build awareness on:

🔹 Early diagnosis and screening
🔹 NiemannPick Disease diagnosis, clinical mgt, referral pathways
🔹 Patient voices
🔹 Diagnosis, screening and support eco system in WB

Our heartfelt thanks to Sanofi for supporting this initiative and to every speaker, doctor and stakeholder who invested their time to learn, collaborate and advance rare disease care.

🧡 With every step like this, we move closer to timely care, equitable access to therapies, and a stronger future for families living with Niemann-Pick and other LSDs in India.

20/11/2025

When the day begins like this…..today is going to be awesome Swasthya Bhawan Kolkata

14/11/2025

This Children’s Day, let’s celebrate them. Let’s stand with them.

Let’s raise awareness so more families get early diagnosis, better care, and access to life-changing treatments.
Because every child, no matter the challenges, deserves a future filled with possibility.

To all the little warriors and their families, you inspire us every day.

Happy Children’s Day!