Rare Diseases

Rare diseases are complex, often genetic conditions that affect a small percentage of the population. These disorders require specialized treatments, many of which are not easily available in all countries. Indian Pharma Network (IPN) is dedicated to providing life-saving medicines for rare diseases through secure and legal supply channels.
We help patients and healthcare professionals access essential rare disease treatments that can improve quality of life and slow disease progression.

Understanding Rare Diseases:
Rare diseases can be genetic, metabolic, autoimmune, or neurological. Many require specialized drugs that are not widely available. Below are some major rare disease categories and their treatments:

• Genetic Disorders: Duchenne Muscular Dystrophy (DMD), Cystic Fibrosis, Fabry Disease
• Neurological Disorders: Spinal Muscular Atrophy (SMA), Neuromyelitis Optica Spectrum Disorder (NMOSD), Epileptic Encephalopathy
• Metabolic Disorders: Mucopolysaccharidosis (MPS), Lysosomal Storage Diseases, Urea Cycle Disorders (UCDs)
• Hematological Disorders: Atypical Hemolytic Uremic Syndrome (aHUS), Hypophosphatasia (HPP)
• Autoimmune & Endocrine Disorders: Thyroid Eye Disease, Acromegaly, Growth Hormone Deficiency

Essential Rare Disease Medicines:
Here are some of the most important rare disease medicines available through Indian Pharma Network:

Ataluren (Translarna):

• Used for: Duchenne Muscular Dystrophy (DMD)
• How it works: Helps produce functional dystrophin protein, slowing muscle degeneration.
• Availability: Oral suspension

Sebelipase Alfa (Kanuma):

• Used for: Lysosomal Acid Lipase Deficiency (LAL-D)
• How it works: Enzyme replacement therapy to prevent fat accumulation in organs.
• Availability: Intravenous infusion

Carglumic Acid (Carbaglu):

• Used for: Urea Cycle Disorders (UCDs)
• How it works: Reduces ammonia levels in the blood.
• Availability: Oral dispersible tablets

Idursulfase (Elaprase):

• Used for: Mucopolysaccharidosis II (Hunter Syndrome)
• How it works: Enzyme replacement therapy to improve organ function.
• Availability: Intravenous infusion

Cerliponase Alfa (Brineura):

• Used for: Batten Disease (CLN2)
• How it works: Slows neurodegeneration in affected patients.
• Availability: Intracerebroventricular infusion

Other Key Rare Disease Medications:

• Uplizna (Inebilizumab-cdon) – Neuromyelitis Optica Spectrum Disorder (NMOSD)
• Strensiq (Asfotase alfa) – Hypophosphatasia (HPP)
• Tavneos (Avacopan) – ANCA-Associated Vasculitis
• Skytrofa (Lonapegsomatropin-tcgd) – Growth Hormone Deficiency
• Nexviazyme (Avalglucosidase alfa-ngpt) – Pompe Disease
• Evkeeza (Evinacumab-dgnb) – Homozygous Familial Hypercholesterolemia (HoFH)
• Nulibry (Fosdenopterin) – MoCD Type A
• Welireg (Belzutifan) – Von Hippel-Lindau (VHL) Disease
• Xiaflex (Collagenase Clostridium Histolyticum) – Dupuytren’s Contracture
• Mepsevii (Vestronidase alfa-vjbk) – MPS VII (Sly Syndrome)
• Symdeko (Tezacaftor/Ivacaftor & Ivacaftor) – Cystic Fibrosis
• Palynziq (Pegvaliase-pqpz) – Phenylketonuria (PKU)
• Onpattro (Patisiran) – Hereditary Transthyretin Amyloidosis (hATTR)
• Galafold (Migalastat) – Fabry Disease
• Diacomit (Stiripentol) – Dravet Syndrome
• Takhzyro (Lanadelumab) – Hereditary Angioedema (HAE)
• Orfadin (Nitisinone) – Hereditary Tyrosinemia Type 1
• Trikafta (Elexacaftor, Tezacaftor, Ivacaftor) – Cystic Fibrosis
• Spinraza (Nusinersen) – Spinal Muscular Atrophy (SMA)
• Amondys 45 (Casimersen) – Duchenne Muscular Dystrophy (DMD)
• Voxzogo (Vosoritide) – Achondroplasia
• Tepezza (Teprotumumab-trbw) – Thyroid Eye Disease
• Isturisa (Osilodrostat) – Cushing’s Disease
• Koselugo (Selumetinib) – Neurofibromatosis Type 1 (NF1)
• VILTEPSO (Viltolarsen) – Duchenne Muscular Dystrophy (DMD)
• XERMELO (Telotristat) – Carcinoid Syndrome Diarrhea
• PARSABIV (Etelcalcetide) – Secondary
• Hyperparathyroidism
• RADICAVA (Edaravone) – Amyotrophic Lateral Sclerosis (ALS)
• RHOPRESSA (Netarsudil) – Glaucoma

Why Choose us for Rare Disease Medicines?

• Access to Hard-to-Find Medicines – We specialize in sourcing rare and orphan drugs.
• Global Reach – Delivering medicines worldwide to patients, hospitals, and resellers.
• Regulatory Compliance – Ensuring safe, legal supply channels for all rare disease treatments.
• Expert Support – Assisting with prescriptions, documentation, and regulatory approvals.
• Secure & Timely Delivery – Cold-chain logistics for temperature-sensitive medicines.