12 discussions tagged #RareDisease.
I'm looking to gather some insights and experiences from folks who have received Mepsevii (10mg/5ml inj) for treating MPS VII. My child's doctor recommended this drug, and I'm curious to hea…
I was recently diagnosed with Fabry disease and started treatment with Fabrazyme 5mg Inj, 1vial. This medicine has been a game-changer for me, but I'm still learning about its side effects a…
I was recently diagnosed with Hereditary Tyrosinemia Type 1 (HT-1), a rare genetic disorder that affects the liver, kidneys, and nervous system. My doctor prescribed Nityr 5mg Tablets, a med…
Vyondys 53 is a revolutionary treatment for Duchenne muscular dystrophy (DMD) in patients who have a specific genetic mutation that can be 'skipped' by this medicine. It works by helping the…
I recently started taking Nityr 2mg Tablets for my hereditary tyrosinemia type 1 (HT-1), and I'm curious to hear from others who are on this medication. It's been a bit of an adjustment with…
I've recently started taking Nitisinone 20mg Capsules to treat my HT-1. The doctor has given me a special diet to go along with it, but I'm curious to know if anyone else has any insight, ex…
I recently started using Adzynma 500iu Inj Kit to manage my congenital thrombotic thrombocytopenic purpura (cTTP). I'm curious to hear from others who have experience with this medication. W…
I recently started using Fyarro 100mg Inj, 1vial to treat my rare type of cancer, PEComa. The medication works by blocking the mTOR protein, which is crucial for cancer cell growth. After a…
I've recently started taking Sohonos 1.5mg Capsules for Fibrodysplasia Ossificans Progressiva (FOP). I'm curious to know if anyone else is taking this medication and how it's working for the…
I've recently started taking Orfadin 5mg Capsules to manage my Hereditary Tyrosinemia Type 1 (HT-1). Along with the medication, I've had to switch to a special low-tyrosine and low-phenylala…
I was recently diagnosed with Acute Hepatic Porphyria (AHP) and my doctor prescribed Givlaari. This is an injectable medicine to treat AHP. The side effects list is a bit scary, and I'm wond…
Hi everyone, I recently started taking Procysbi 25mg Capsules to manage nephropathic cystinosis. I'm curious to hear from others who have experience with this medication. What side effects h…