News

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.

Legislators and a libertarian think-tank have flaunted the merits of the Right-to-Try experimental drug policy. But physicians question its value and the message it sends to patients.

The study adds new data in the developing world for the aging HIV population, suggesting that long-term cART exposure and HIV duration have harmful effects.

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.

Physical and social improvements were observed in the 8-month visit, but importantly, were sustained in the 3-year follow-up period.

A component in inflammatory pathogenesis including airway inflammation could serve as a biomarker in non-eosinophilic asthma.

Although popular, organic light-emitting sleep masks have been reported to be ineffective in reducing disease progression for patients with diabetic macular edema.

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.

Study results showed significant HbA1c reduction when Bydureon was added to insulin glargine therapy versus insulin glargine alone.

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.

In a first-time issuance, all food products containing powdered kratom from Triangle Pharmanaturals, have been ordered to be recalled due to samples containing salmonella bacteria.

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

The decision was made with data from the REDUCE study, which showed a 77% reduction in recurrent ischemic stroke in the patients who underwent PFO closure with the device.

Researchers observed that prescribing rates were cut by about 6% in at-risk populations from states with legalization laws in place.

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.

The state of your manicure could be contributing to infection transmission. ​​​​​

A study has shown that those who underestimated their prescription rates decreased opioid prescriptions by 2.2 patients more over the next 12 months after being notified of their data.

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.

The Pfizer molecule previously received a Fast Track designation by the FDA in June 2017.

The 27-year Navy veteran will assume clinical and administrative roles with the country's largest clinical research-focused hospital.

The claim that users’ HIV status being shared with 2 other companies calls into question the company's users' privacy policy.

"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.

The FDA is requesting that additional clinical trials, along with a bioavailability study, are needed to resubmit ALKS 5461.

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