Rare Warriors: Organization Leading the Fight Against Rare Disease
Friday, February 28, 2025 ●10 min read
-By VOH Team
The Indian Organization for Rare Diseases (IORD)
The Indian Organization for Rare Diseases (IORD) is a registered non-profit and a Ministry of Corporate Affairs-recognized CSR Implementing Agency advocating for rare disease patients in India. It envisions a future where these patients receive equal healthcare opportunities and focuses on policy development, awareness, and patient support. IORD educates medical professionals, assists in diagnosis, and facilitates treatment access. It also promotes research and global collaborations to improve care. Addressing awareness gaps, social stigma, and delayed diagnoses, IORD actively works to enhance healthcare policies and ensure better resources for rare disease patients across India.
Dystrophy Annihilation Research Trust [DART]
Dystrophy Annihilation Research Trust (DART) is India’s first research lab dedicated to Duchenne Muscular Dystrophy (DMD). Founded by RS Anand in 2012, it focuses on genetic-level treatments to slow or reverse DMD progression. The organization provides patient support, medical advisory services, rehabilitation assistance, and counseling for families. DART also raises awareness, promotes inclusion in education and employment, and collaborates with NGOs. Its R&D lab offers much-needed research within India, giving hope to affected families by ensuring access to critical interventions.
Cure SMA Foundation
Cure SMA India is a nonprofit organization dedicated to supporting individuals with Spinal Muscular Atrophy (SMA), a rare genetic disorder causing progressive muscle weakness due to motor neuron degeneration. The NGO focuses on patient advocacy, early diagnosis, and access to life-saving treatments, without specifying treatment names. It provides medical assistance, rehabilitation support, and family resources while collaborating with researchers and policymakers to improve treatment availability and affordability. Through awareness campaigns and community engagement, Cure SMA India empowers individuals affected by SMA and advocates for improved healthcare policies in India
The Lysosomal Storage Disorders Support Society (LSD) The Lysosomal Storage Disorders Support Society (LSD) , established in 2010, is India's first and leading patient advocacy group dedicated to Lysosomal Storage Disorders (LSDs). A pan-India non-profit organization, it is managed by patients and their parents/caregivers, focusing on creating awareness among the public, physicians, and policymakers. LSDSS has achieved significant milestones by facilitating treatment options through international charitable access programs and state healthcare budgets. They have also played a pivotal role in the formulation of India's National Policy for Rare Diseases (NPRD). Their work areas include patient support, advocacy, awareness campaigns, and collaboration with national and international organizations to strengthen the patient voice and improve LSD awareness.
The Bharath MD Foundation
The Bharath MD Foundation, founded by mothers of children with Duchenne Muscular Dystrophy (DMD), is dedicated to support, advocacy, and awareness for affected families. It provides free genetic testing, medical and rehabilitation support, and educational resources to empower individuals with DMD. The foundation collaborates with researchers and policymakers to enhance treatment access and drive policy changes. Key initiatives focus on awareness campaigns, research partnerships, and expanding medical services. With a vision for improved care and inclusion, the organization continues to champion hope, resilience, and change for the DMD community in India.
The Atypical HUS foundation
aHUS India is a dedicated support network for individuals affected by Atypical Hemolytic Uremic Syndrome (aHUS) in India. The organization focuses on patient advocacy, early diagnosis, treatment access, and community support. Recently, aHUS India has been working to connect Indian patients with global research efforts, ensuring access to the latest advancements in complement-inhibitor therapies. It has also been advocating for insurance coverage and government policy inclusion for aHUS treatments. Through awareness campaigns, patient guidance programs, and expert collaborations, aHUS India is striving to improve the quality of life for those battling this rare and life-threatening disease.
Mobility in Dystrophy (MIND) Trust
Mobility in Dystrophy (MIND) Trust, founded in 2017, supports individuals in Kerala with Muscular Dystrophy (MD) and Spinal Muscular Atrophy (SMA) through 18 projects focused on awareness, education, skill development, rehabilitation, and research. Recognized for its efforts, MIND Trust received Kerala’s Best NGO Award (2021), N.S Hema Memorial 3rd position (2023), and the 12th FESCA Award (2024). The organization is dedicated to realizing ORIDAM, a holistic center providing rehabilitation, residential care, education, employment, and research to empower individuals with MD and SMA, fostering independence and self-sustainability while advancing innovative approaches for better management and support.
DATRI Blood Stem Cell Donors Registry
DATRI Blood Stem Cell Donors Registry is a not-for-profit organization committed to helping patients with life-threatening blood disorders - such as leukemia, thalassemia, lymphoma, and aplastic anemia - by facilitating life-saving blood stem cell transplants.DATRI actively identifies and registers potential donors by raising awareness about stem cell donation. Through collaborations with hospitals, medical institutions, colleges, and corporates, it works to build a diverse and accessible donor database for patients in need.With a registry of over 5.7 lakh donors, DATRI has successfully facilitated more than 1,530 stem cell donations, offering patients battling blood disorders a second chance at life.As India’s largest unrelated blood stem cell donor registry, DATRI plays a pivotal role in saving lives through donation, advocacy, and continuous efforts to expand its life-saving impact.
Indo US Organization for Rare Diseases
"Indo US Organization for Rare Diseases is a non-profit organization dedicated to rare disease research, advocacy, and collaboration between India and the U.S. Their mission is to educate, empower, and advocate for patients globally, fostering diversity, equity, and inclusion in research. They focus on patient-centric education, awareness, training, and engagement aligned with the FDA’s patient-focused drug development paradigm. Indo US organization for rare diseases promotes global diversity in clinical trials, improves patient access to research, and collaborates on public health policies. By bridging stakeholders from India and the U.S., they aim to enhance patient outcomes, accelerate diagnostics and therapies, and address challenges faced by rare disease communities.”
The Indian Society for Inborn Errors of Metabolism (ISIEM)
The Indian Society for Inborn Errors of Metabolism (ISIEM), conceived in 2005 and registered as a society in 2012, is dedicated to improving awareness, diagnosis, and management of inherited metabolic disorders in India. Their core work areas include research, patient advocacy, physician training, and policy advocacy for better healthcare access. ISIEM organizes national and international conferences, workshops, and training programs to educate healthcare professionals on inborn errors of metabolism (IEM). ISIEM also publishes clinical guidelines & protocols on management , collaborates on newborn screening programs, and works towards making diagnostic and therapeutic facilities more accessible. ISIEM is known for pioneering efforts in metabolic disorder awareness and strengthening India's rare disease ecosystem.
Cystic Fibrosis India (CFI)
Cystic Fibrosis India (CFI) is committed to improving the lives of individuals with Cystic Fibrosis (CF) through advocacy, support, and awareness. The organization works to ensure early diagnosis, access to treatment, and specialized care for CF patients across India. CFI provides medical assistance, nutritional guidance, and rehabilitation support, helping families navigate the challenges of this rare genetic disorder. By collaborating with doctors, researchers, and policymakers, CFI advocates for better healthcare infrastructure and treatment availability. Through education, outreach programs, and community initiatives, the foundation empowers patients and caregivers, striving to build a more informed and supportive environment for CF care.
Hereditary Angioedema Society of India (HAESI)
The Hereditary Angioedema Society of India (HAESI) (https://www.haesi.in/) is a nonprofit organisation dedicated to increasing awareness and to promote knowledge about HAE amongst physicians; to improve quality of life of HAE patients and their families and bring better treatment options in India. The society has actively collaborated with global HAE networks over the last 4 years. HAESI has organized several specialist training workshops to improve early detection and has been lobbying for the inclusion of HAE therapies in national health programs. The society also provides direct patient support, emergency treatment guidance, and counseling services. Through these efforts, HAESI is bridging the gap between medical advancements and patient accessibility in India.
MSN Laboratories
MSN Laboratories has launched RADIUM, a dedicated division focused on rare diseases and unmet medical needs. As a global pharmaceutical leader, MSN offers over 60 solutions for rare conditions, reinforcing its commitment to innovation. With 25 advanced manufacturing facilities across India and the USA and Asia’s largest single-site integrated R&D center, MSN drives cutting-edge research for better medicines. The company’s diverse portfolio spans 475+ APIs and 350+ formulations across 40 therapeutic areas, serving over 50 million patients in 100+ countries. Through RADIUM, MSN continues to bring hope to rare disease patients worldwide, advancing access to life-saving treatments.
Akums Drugs and Pharmaceuticals
Akums Drugs and Pharmaceuticals, India’s largest CDMO, is revolutionizing rare disease treatment with innovation and accessibility. In line with its Rashtra Pratham approach, Akums has introduced a Room Temperature Stable Hydroxyurea Oral Suspension for Sickle Cell Disease (SCD), significantly improving affordability and access. Unlike global alternatives requiring cold storage and costing INR 77,000, Akums’ patented 2024 formulation is stable at room temperature and priced at just 1% of the global cost. With expertise across pharmaceuticals and advanced R&D, Akums continues to pioneer solutions for rare diseases, reinforcing its commitment to addressing critical healthcare challenges in India and beyond.
CENTOGENE
With over 70 million people in India affected by rare diseases, timely diagnosis remains a critical challenge. CENTOGENE envisions reducing this diagnostic odyssey of undiagnosed and misdiagnosed cases through technological advancements. CENTOGENE is at the forefront of rare disease diagnostics, aiming to cure 100 rare diseases by 2031 through AI-driven bioinformatics, expansive data access, and continuous variant reclassification. Leveraging its Biodatabank - the world’s largest repository of real-world data on rare and neurodegenerative diseases- CENTOGENE is uncovering novel Gene-Disease Relationships. Additionally, early 2024 collaborations with the Laboratory of Human Genetics of Infectious Diseases and the Critical Path Institute have accelerated drug discovery. CENTOGENE’s groundbreaking research indicates that approximately 15% of Parkinson’s cases have a genetic link, reinforcing the need for genetic testing in patient care.
Disclaimer:
The information presented in this listicle, Rare Warriors: Organization Leading the Fight Against Rare Disease, is intended for informational purposes only. While we strive to provide accurate and up-to-date details about these organizations and their initiatives, we do not endorse or guarantee the efficacy of any treatments, services, or policies mentioned. Readers are encouraged to verify information through official sources and consult healthcare professionals for medical advice, diagnosis, or treatment.
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