RDR Alert®

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.

Survey results from the IPA/Erasmus MC Pompe survey investigated whether ERT can reduce Pompe disease patients’ risk of needing either a wheelchair, respirator, or both.

Results from an online, self-administered survey showed a mostly consistent and unified response in pulmonologist treatment responses.

An analysis of over 1,200 patients from pivotal studies finds that Esbriet helped blunt seasonal peaks in respiratory hospitalizations among patients with idiopathic pulmonary fibrosis.

Supported by positive data from an ongoing Phase 2/3 study, bluebird bio’s Lenti-D has been granted Breakthrough Therapy designation by the US FDA for the treatment of patients with cerebral adrenoleukodystrophy.

New data presented yesterday at the American Thoracic Society’s 2018 annual conference suggest that treatment with nintedanib (Ofev) is associated with a reduced risk of death in patients with idiopathic pulmonary fibrosis.

At NORD’s 2018 Rare Impact Awards, Rare Disease Report® sat down with Estelle Benson, founder of GBS|CIDP Foundation International, to discuss what the organization is doing to fight GBS.

Yesterday, UroGen Pharma Ltd released positive data from its interim analysis of the ongoing pivotal Phase 3 OLYMPUS clinical trial of UGN-101 (MitoGel) for the non-surgical treatment of low-grade upper tract urothelial cancer (UTUC).