Positive Results in Trials of First Oral Investigational Drug for Acromegaly

Positive Results in Trials of First Oral Investigational Drug for Acromegaly

Signs and Symptoms of Fabry Disease, Featuring Nicola Longo, MD (Podcast)

Signs and Symptoms of Fabry Disease, Featuring Nicola Longo, MD (Podcast)

Fabry Disease From a Patient’s Perspective, Featuring Maya Kineen (Podcast)

Fabry Disease From a Patient’s Perspective, Featuring Maya Kineen (Podcast)

Fabry Disease Overview, Featuring William Burns, MD (Podcast)

Fabry Disease Overview, Featuring William Burns, MD (Podcast)

Diagnosing Fabry Disease

Diagnosing Fabry Disease

Fabry Disease and Immunogenicity

Fabry Disease and Immunogenicity

Managing Fabry Disease

Managing Fabry Disease

Treatment Plans for Fabry Disease

Treatment Plans for Fabry Disease

Fabry Disease Patient Story

Fabry Disease Patient Story

Fabry Disease Patient Journey

Fabry Disease Patient Journey

Investigational Drug for Hereditary ATTR Amyloidosis

Investigational Drug for Hereditary ATTR Amyloidosis

Patient Experience With Myasthenia Gravis

Patient Experience With Myasthenia Gravis

Results from Fabry Patient Survey

Results from Fabry Patient Survey

How Rare Diseases Impact Daily Life: Scleroderma

How Rare Diseases Impact Daily Life: Scleroderma

Phase 1 Investigational Drug for Rare Blood Cancers

Phase 1 Investigational Drug for Rare Blood Cancers

Patient Perspective: NMOSD Diagnosis and Finding Support

Patient Perspective: NMOSD Diagnosis and Finding Support

Living With Myasthenia Gravis

Living With Myasthenia Gravis

Positive Preliminary Results for Gene Therapy in Auditory Neuropathy Spectrum Disorder

Positive Preliminary Results for Gene Therapy in Auditory Neuropathy Spectrum Disorder

Fibrodysplasia Ossificans Progressiva: Overview and Advocacy

Fibrodysplasia Ossificans Progressiva: Overview and Advocacy

Imperfect JOY: Capturing the Lives of Patients With Rare Diseases

Imperfect JOY: Capturing the Lives of Patients With Rare Diseases

The Cushing's Support and Research Foundation

The Cushing's Support and Research Foundation

The Diagnostic Journey of Cushing's Patients

The Diagnostic Journey of Cushing's Patients

Raising Awareness for Fibrodysplasia Ossificans Progressiva

Raising Awareness for Fibrodysplasia Ossificans Progressiva

Being an Advocate for Yourself and Others

Being an Advocate for Yourself and Others

Fulfilling the Unmet Needs of Sarcoidosis Patients

Fulfilling the Unmet Needs of Sarcoidosis Patients

Lenmeldy For Metachromatic Leukodystrophy (MLD): Challenges of Gaining RUSP Approval

Lenmeldy For Metachromatic Leukodystrophy (MLD): Challenges of Gaining RUSP Approval

Challenges of the FDA in Regards to Rare Diseases

Challenges of the FDA in Regards to Rare Diseases

Gene Therapy for Metachromatic Leukodystrophy (MLD): Addressing the Cost Issue

Gene Therapy for Metachromatic Leukodystrophy (MLD): Addressing the Cost Issue

Expanded Access Programs and Barth Syndrome Drug Candidate

Expanded Access Programs and Barth Syndrome Drug Candidate

Addressing Mental Health in Patients with Rare Diseases

Addressing Mental Health in Patients with Rare Diseases