RDR Alert®

First-in-man phase 1 trial meets its primary endpoints by demonstrating safety and immunogenicity of a mutation-specific IDH1R132H peptide vaccine.

To date, OPTIMISMM is the only phase 3 study in early RRMM to report a significantly and clinically meaningful PFS improvement in LEN-exposed patients.

Prospective genomics can inform subsequent clinical trial analysis aiming at matching outcome with tumor genotyping.

Express Scripts announces a new Rare Conditions Care Value program for rare disease patients with introduction support service of Second Opinion in partnership with PinnacleCare.

Saby George, MD, FACP, and Dena Battle, president of nonprofit patient advocacy group, KCCure, discussapproval of Opdivo (nivolumab) and Yervoy (ipilimumab) therpay combination for kidney cancer.

Researchers find that a specially formulated antioxidant-enriched supplement may decrease respiratory illnesses in patients with cystic fibrosis.

The US FDA has granted Genea Biocells Orphan Drug Designation to its product, GBC0905, for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

Upon unearthing rheumatoid arthritis’ epigenome, researchers at University of California San Diego School of Medicine and the Icahn School of Medicine at Mount Sinai have also discovered an overlap between the causes of rheumatoid arthritis and Huntington’s disease.

The FDA approved the first artificial iris for both pediatric and adult patients with aniridia who have an iris that is completely missing or damaged.

Marijke van den Berg, MD, PhD, Alok Srivastava, MD, and Glenn Pierce, MD, PhD discuss gene therapy in hemophilia.

In an exclusive interview, Cary O. Harding, MD, provides Rare Disease Report® with insight into pegvaliase-pqpz and its recent approval by the FDA to treat patients with PKU.

The FDA has accepted its supplemental New Drug Application (sNDA) and granted Priority Review designation to Promacta (eltrombopag) in combination with standard immunosuppressive therapy (IST) for first-line treatment of severe aplastic anemia (SAA).

The US FDA has granted Astellas Pharma Inc priority review for gilteritinib for refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test.

The Nipah virus outbreak in India's southern state of Kerala may have spread to the neighboring state of Karnataka.

The FDA has accepted the NDA and granted Priority Review to Firdapse for the treatment of LEMS.

FDA grants larotrectinib New Drug Application and Priority Review for the treatment of locally advanced or metastatic solid tumors containing an NTRK gene fusion on both adult and pediatric patients.

By adjusting protein levels that impact an important biological pathway, researchers improve heart function in mice with HD, shedding light on the biology of the disease.

A recent study sheds light on how the bacteria responsible for necrotizing fasciitis invades the body and explores the role Botox might be able to play in stopping these infections.

In an article recently published by the Institute of Cancer Research, genetic testing was indicated to result in life-extending treatment for incurable pediatric brain tumors.

Facial analysis technology can help identify the most characteristic and relevant facial features found in Williams-Beuren syndrome (WBS) in diverse populations.

Burosumab found to improve outcomes in children with x-linked hypophosphatemia in phase 2 study.

Daniel de Boer, CEO of ProQR, Rare Disease Report ® discusses the company’s new RNA therapy, QR-313, for the treatment of dystrophic epidermolysis bullosa (DEB).

The FDA has approved pegvaliase-pqpz for the treatment of adults with PKU.

iLLUMINATE trial meets primary endpoint of PFS in patients with previously untreated chronic lymphocytic leukemia compared with chlorambucil plus obinutuzumab.

In a recent study, hematologists were dubbed pivotal in finding and treating Gaucher disease Type 1.

Christopher-Paul Milne, research associate professor, director of research at Tufts CSDD at Tufts University School of Medicine and the analysis conductor of the study, further explains orphan drug development.

This morning, Pfizer announced that its investigational therapy for the treatment of patients with transthyretin (TTR) cardiomyopathy, tafamidis, received Breakthrough Therapy designation from the US FDA.

Until her death in April, Tannehill served as the Associate Professor of Education at Robert Morris University in Pittsburgh, PA. This story is about her journey to her AL Amyloidosis diagnosis.

Congress has voted to pass the Right to Try bill which will provide terminally ill patients with the ability to seek out experimental therapies not yet approved by the FDA.

Two early-stage studies of gene therapies from Pfizer, Spark Therapeutics, and BioMarin Pharmaceutical indicate efficacy for hemophilia A and B treatment.