RDR Alert®

The Phase 2 CARDINAL study is evaluating bardoxolone methyl (bardoxolone) in patients with chronic kidney disease because of Alport syndrome.

Results from a new study led by researchers in the CCR at the NCI could potentially explain why some patients with diffuse large B-cell lymphoma respond to treatment and others don’t.

FDA approves Phase 2 study of KD025, a a possible treatment for graft-versus-host disease (GVHD).

Jersey Mike’s Raises $377,000 for Make-A-Wish New Jersey.

CEO of Krystal Biotech discusses first topical gene therapy for dystrophic epidermolysis bullosa.

Results from a study published in Nature Communications offer potential treatment targets for melorheostosis, often referred to as “dripping candle wax” bone disease.

The self-administration of pnf C1-INH (Berinert) proves effective for patients with hereditary angioedema with C1 inhibitor deficiency.

Data from the article, “Fabry disease in the Spanish population: oberservational study with detection of 77 patients,” suggests that enzymatic screening for Fabry disease in risk populations assisted in identifying undiagnosed patients.

This morning, it was announced that everolimus (Afinitor DISPERZ) has become the first therapy to be specifically approved by the U.S. FDA to treat patients aged 2 years and older with tuberous sclerosis complex (TSC)-associated partial-onset seizures.

Higher-quality epidemiological data is needed in all 4 subtypes of Sanfilippo syndrome for better disease research and management.

André the Giant, examines the life and work history of the WWE Hall of Famer. Unbeknownst to many, his size was the result of gigantism, a disease caused by excess growth hormone. The condition led to his eventual diagnosis with acromegaly, a rare endocrine disorder.

In a recent interview with Dr Steven Freedman, Rare Disease Report asked for the specifics on familial chlyomicronemia syndrome (FCS).

A new provisional patent was filed by Nutra Pharma Corporation to protect the intellectual property surrounding their development of a drug intended to treat amyotrophic lateral sclerosis.

Early this morning, Reviva Therapeutics announced that the U.S. Food and Drug Administration has granted orphan drug designation to its clinical stage drug candidate RP5063, in development for the treatment of idiopathic pulmonary fibrosis.

FDA grants orphan drug designation to elamipretide for the treatment of Barth syndrome.

At the most recent American Lung Association LUNGFORCE expo, RDR sat down with Albert Rizzo, M.D. of the Christiana Care Pulmonary and Critical Care Medicine Section to discuss previous, current and emerging therapies for idiopathic pulmonary fibrosis.

This morning, AbbVie revealed positive top-line results from the Phase 3 SELECT-COMPARE clinical trial evaluating upadacitinib in rheumatoid arthritis.

Novartis and AveXis merge spells opportunity for SMA research.

When taken early in a pregnancy, mifepristone can end a pregnancy that is less than 49 days along. Another notable trait of the drug, though, is its ability to control the high blood sugar in patients with Cushing syndrome who have type 2 diabetes and have either failed surgery or are not candidates for it.

Rare Disease Report tries to keep its audience at the forefront with the latest news from the hematological and oncological spaces. This new infographic provides 5 little-known facts about MPNs that might not necessarily be covered in news articles from RDR or other media outlets.

Rhizen Pharmaceuticals SA's product, Tenalisib (RP6530), is granted orphan drug designation from the FDA for the treatment of CTCL.

Tarekegn Geberhiwot, MD, of the Institute of Metabolism and Systems Research and colleagues had an article published in the Orphanet Journal of Rare Diseases, outlining the development of clinical management recommendations for Niemann-Pick disease type C.

QurAlis Corporation joins forces with investors MP Healthcare Venture Management (MPH), Amgen Ventures, and Alexandria Venture Investments to find a cure for ALS.

Molecular characteristics that link the genomic profiles of squamous cell carcinomas from 5 areas of the body have been uncovered by a team of researchers supported by the NIH.

Phase 2 Study with Lanifibranor on Non-Alcoholic Fatty Liver Disease in Patients with Type 2 Diabetes Commences.

The FDA granted orphan drug designation to fluasterone (ST-002) for the treatment of Cushing’s syndrome.

Treatment with nivolumab, followed by ipilumab, produced significantly higher overall survival and objective response rates than sunitinib alone among intermediate- and poor-risk patients with previously untreated advanced renal-cell carcinoma.

Researchers Clive Svendsen, PhD and Samuel Sances of the Cedars-Sinai Board of Governors Regenerative Medicine Institute in Los Angeles note that spinal neuron development can be initiated by the human brain’s tiniest blood vessels.

Preclinical murine study data from Sangamo Therapeutics’ in vivo genome editing program for MPS II provided proof of concept for development of its product candidate SB-913.

PTC Therapeutics, Inc. and the CDHI Foundation Inc. are partnering for a research collaboration that will hopefully advance the former’s Huntington’s disease program.