RDR Alert®

Califf joins HCPLive to share perspective on AI, gene and cell therapy development, COVID-19, food and substance regulation, maternal and infant health, weight loss intervention, and more.

Phase 3 data presented at ASH 2023 show the gene therapy significantly reduced annualized bleeding rates and the number of total bleeds.

Two experts describe the correlation between the severity of arginine deficiency and worse clinical outcomes among hospitalized children with sickle cell disease experiencing vaso-occlusive pain episodes.

For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.

The approval is the first for a systemic therapy to treat desmoid tumors, offering patients an alternative to surgery.

Safer discusses how symptoms and quality of life often go unaddressed in the few clinician-patient interactions about primary sclerosing cholangitis.

Pooled data from The Liver Meeting showed odevixibat sustains itch and serum bile acid benefits in patients with Alagille syndrome. An investigators shares what he wants to learn next.

New pooled phase 3 and extension trial data show odevixibat continues to improve disease management and itch reduction in children with Alagille syndrome.

The expanded Indication now includes children of all ages with achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature.

Maralixibat and odevixibat have been "game changers" for their benefit in pruritus, and clinicians are interested to learn more about their long-term hepatic benefit.

Though a regimen of steroids and immunosuppressants are well proven, alternative options may be necessary.

Particularly in children, it's critical that referring physicians keep an awareness for clear symptoms and signs of autoimmune liver disease.

The author of the largest clinical trial for pediatric PFIC to date discusses the various benefit maralixibat provides to disease managementr.

Pooled phase 3 data presented at NASPGHAN 2023 support the benefit-risk profile of the IBAT inhibtor in treating the rare liver disease.

Announced by Ionis Pharmaceuticals, topline results show olezarsen met the study's primary endpoint with a statistically significant reduction in triglyceride levels versus placebo.

Crinetics announced findings from the PATHFNDR-1 trial showing the investigative oral daily drug may provide patients with the rare disease an alternative from recurring injection therapy.

The FDA approval of pozelimab (Veopoz) makes it the first and only treatment indicated for children and adults with CHAPLE disease.

Phase 3 data show a significant clinical profile associated with durable reductions in IgA nephropathy biomarkers among patients treated with Nefecon.

Granted to Ipsen, the breakthrough treatment reduces new, abnormal bone growth in people living with the ultra-rare bone disease.

Dr. Mikkael Sekeres discusses the approval of quizartinib (Vanflyta) and how it impacts the treatment of acute myeloid leukemia.

Reports of a half-dozen confirmed cases of the rare vision-threatening condition has spurred conversation around the first-in-class geographic atrophy drug.

This approval is the first for an FLT3 inhibitor and it's specifically indicated for patients who have FLT3-ITD.

A year of research emphasizes the significance of collaboration, standardized approaches, and targeted therapies in rare pulmonary diseases.

If approved, OFEV (nintedanib) would be the first and only approved treatment for children and adolescents aged 6-17 years old with fibrosing interstitial lung disease.

New Study Reveals Efficacy of Bevacizumab in Treating Severe Cases of Pulmonary Papillomatosis.

Investigators aimed to support extending the US FDA labeling of Xembify to include the biweekly dosing option, therefore, providing greater flexibility and convenience for patients managing their immunodeficiencies.

The findings show liver complications and cirrhosis as major causes of morbidity in patients with SCD, with implications for treatment and care.

Experts emphasize the fundamental principles of integrative care, the success seen in treating rare diseases, and how it can be applied to all chronic diseases.

Efanesoctocog alfa defined a new therapy class by the US Food and Drug Administration (FDA) as the first and best-in-class high-sustained FVIII therapy.

Efanesoctocog alfa demonstrates promising results in new phase 3 data emphasizing the drug's potential to address unmet needs within the hemophilia community.