RDR Alert®

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.

The time to revisit treatment approaches for gout is now.

Parents of children with the rare disease Congenital Muscular Dystrophy are spending the next seven weeks preparing for the 5th Annual Million Dollar Bike Ride, to be held in Philadelphia on May 20th.

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.

Results from a recent study found that the mutated huntingtin gene in patients with HD generates a class of small molecules that are highly toxic to cancer cells, but not healthy cells.

A study published in the journal eLife suggests that blocking an enzyme could reduce levels of the mutant huntingtin (mHTT) protein.

Insmed Incorporated announced that it has submitted its New Drug Application for amikacin liposome inhalation suspension (ALIS) to the U.S. FDA.

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to blinatumomab (Blincyto) for the treatment of adults and children with B-cell precursor ALL who are in remission, but still have minimal residual disease.

In the ATTR-ACT study, tafamidis exhibited a statistically significant reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations in transthyretin cardiomyopathy patients.

Catalyst Pharmaceuticals announces its submission of an NDA to the FDA for amifampridine phosphate (Firdapse) for the treatment of LEMS.

A new study concludes that, among patients with AML, the detection of molecular minimal residual disease during complete remission had significant independent prognostic value with respect to relapse and survival rates.

The FDA granted orphan drug designation to Scholar Rock for its lead antibody product candidate, SRK-015, for the treatment of spinal muscular strophy (SMA).

Clinical data from an ongoing Phase 2 study of PBI-4050 shows that the drug provides a clear clinical benefit in patients with Alstrom syndrome.

Phase 3 of the NEWTON 2 study of EG-1962 in aSAH has been terminated since it is unlikely to meet its primary endpoint.

The FDA granted Cynata’s Therapuetics Limited (ASX:CYP) orphan-drug designation for CYP-001, which is the company’s potential treatment for acute graft versus host disease (GvHD).

Results from a trial sponsored by the National Cancer Institute demonstrate that sorafenib tosylate (Nexavar) extended progression-free survival in patients with desmoid tumors or aggressive fibromatosis.

The FDA granted GeneTx Biotherapeutics LLC orphan-drug designation for GTX-101, the company’s potential treatment for Angelman syndrome.

At the 16th International Symposium on Amyloidosis, Alnylam presented results from the APOLLO Phase 3 study of patisiran, which targets transthyretin in hATTR amyloidosis.

The U.S. FDA announced that Breakthrough Therapy Designation had been granted to Fennec Pharmaceuticals for its unique formulation of cisplatin and sodium thiosulfate (STS).

Eiger announced first patient treated in the PREVENT study, a phase 2, multicenter study of subcutaneous (SC) exendin 9-39 in post-bariatric surgical patients who experience PBH.