RDR Alert®

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.

Using MRIs and computer models, researchers from the University of North Carolina School of Medicine were able to prove that babies who develop the fragile X syndrome have less white matter circuitry than infants who did not.

The FDA has accepted BLA for hairy cell leukemia treatment.

Altemia, a new treatment for sickle cell disease (SCD), was granted orphan drug designation.

REVERSE Phase III clinical trial shows efficacy and safety of GS010 in patients with LHON.

Topline data from Rigel Pharmaceuticals’ proof-of-concept Phase 2 study of fostamatinib in patients with IgA nephropathy, or Berger’s disease, were announced today.

Phase 3 OLYMPUS clinical trial releases interim analysis of UGN-101 (MitoGel) for low-grade UTUC.

BPN14770, in development for the treatment of Fragile X syndrome, was granted Orphan Drug Designation by the U.S. Food and Drug Administration this morning.

FDA grants orphan drug designation to PharmAbcine Inc for TTAC-0001, the company’s leading clinical compound, for treatment of GBM.

The first patient has been dosed in MyoKardia’s Phase 2 MAVERICK-HCM clinical trial of mavacamten in symptomatic non-obstructive hypertrophic cardiomyopathy patients.

"A perspective on ‘cure’ for Rett syndrome” concluded that therapies should focus on improving patients’ quality of life, and expectations for a radical, rational treatment should not be “inflated beyond cautious optimism.

Results from a study published have shown consistency with previous data, concluding that dual targeting is effective in mantle cell lymphoma.

FDA Commissioner, Scott Gottlieb, MD released a statement this morning, stressing the FDA’s intention to incorporate the patient experience into the regulatory authority’s benefit-risk assessments.