Articles

Podcasts

Videos

Patients with sarcoidosis who practiced online mindfulness-based cognitive therapy (eMBCT) showed improvements in fatigue, anxiety, depression, and health status.

Sarcoidosis-Associated Symptoms Improve With Mindfulness-Based Cognitive Therapy

The BLA was supported by positive data from 54 patients with hemophilia B in the pivotal phase 3 HOPE-B trial.

FDA Approves First Gene Therapy for Hemophilia B

Therapeutic candidate Sparsentan could expand treatment options for 2 rare kidney diseases. Dr. Jula Inrig expands on the potential for the FSGs patient population.

Sparsentan Could Address Unmet Need in Focal Segmental Glomerulosclerosis Population

Dr. Jula Inrig explains the mechanism of action for the Dual Endothelin Angiotensin Receptor Antagonist (DEARA) investigational candidate Sparsentan, for rare kidney diseases.

The Future of Sparsentan According to Travere Therapeutics Chief Medical Officer

Nonprofit organizations combined efforts to plan a meeting to inform and educate the FDA and key stakeholders about the experience of caregivers and patients with Phelan-McDermid syndrome (PMS).

Caregivers of Individuals With Phelan-McDermid Syndrome and FDA Discuss Treatment Potential

Dr. Jula Inrig highlights the key data presented at ASN Kidney Week on investigational candidate Sparsentan for IgA nephropathy.

Jula Inrig, MD: New Data on the Burden of Immunoglobulin A Nephropathy

Aside from the inability to fulfill the site inspection, there was no other reason for the FDA deferral. The approval of the combination drug candidate's 2 components are still expected.

FDA Defers Action on Novel Pompe Disease Therapy Cipaglucosidase Alfa, AT-GAA Decision

The Pompe disease community is anticipating the pivotal FDA decision for AT-GAA, a combination therapy for late-onset Pompe disease (LOPD) that has the potential to expand the armamentarium of physicians.

Rare Disease Report Podcast: FDA Reviews Combination Therapy for Pompe Disease

Dupilumab represents the first ever approved treatment for EoE.

Evan Dellon, MD, MPH: New Late-Breaking Data on Dupilumab for EoE

Iptacopan has the potential to address an unmet need in PNH by significantly increasing hemoglobin levels in patients who remain anemic, fatigued and dependent on blood transfusions after anti-C5 treatment.

Paroxysmal Nocturnal Hemoglobinuria Drug Candidate Demonstrates Superiority to Anti-C5 Therapies

Sickle Cell

Sickle Cell condition center page, resources on the topics of medical news and expert insight into sickle cell disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on sickle cell research, treatment, and drug development.

Amyloidosis

The HCPLive amyloidosis page is a resource for medical news and expert insights on the rare disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for amyloid buildup, and more.

Duchenne Muscular Dystrophy

On the HCPLive Duchenne muscular dystrophy page, resources on the topics of medical news and expert insight into DMD can be found. Content includes articles, interviews, videos, podcasts, and breaking news on the rare disease, and more.

Huntington Disease

On the HCPLive Huntington disease page, resources on the topics of medical news and expert insight into HD can be found. Content includes articles, interviews, videos, podcasts, and breaking news on Huntington's chorea, and more.

Lysosomal Storage Disorders

Lysosomal storage disorders page, resources on the topics of medical news and expert insight into the rare diseases can be found. Content includes articles, interviews, videos, podcasts, and breaking news on lysosomal disorder disease research, treatment, and drug development.

The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.

RDR Alert®

Latest News