Why Ask, ‘Why Me?’ Don’t Play The Blame Game with Yourself

Nonprofit Group Works to Raise Rare Disease Awareness in India

With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…

Other Diseases and Treatment Reactions Common in Myasthenia Gravis Patients, Study in India Finds

Almost 90% of patients diagnosed with myasthenia gravis develop other diseases, or comorbidities, while about 70% have treatment-related complications, a review study from India reports. These additional disorders may contribute to a poorer outcome for these people, making early recognition and management of comorbidities essential for improving patient care and overall…

Tell Me Why We Need to Talk About Organ Donation

Recently, I attended an event that reminded me of the importance of speaking up, letting others into your world, and starting a conversation to create awareness. The event was organized by the South African nonprofit organization Tell, which stands for “Transplant Education for Living Legacies.” It aims to encourage…

Ra Pharma Set to Launch Phase 3 Trial Testing Zilucoplan on Patients with Generalized Myasthenia Gravis

Ra Pharmaceuticals is preparing to launch a Phase 3 clinical trial to assess the therapeutic potential of zilucoplan for the treatment of patients with generalized myasthenia gravis (gMG). This comes after the company’s completion of End-of-Phase 2 interactions with the U.S. Food and Drug Administration (FDA). The 12-week study — a single, pivotal,…

With Rare Disease, You Must Be Your Own Advocate

Hearing the words “you have a rare disease” is not something you ever expect. Nor is it anything you could ever really prepare for. But unfortunately, it does happen — and it may have happened to you if you are reading this. Going to a general practitioner when you have…