News

In an interview with Rare Disease Report, Mathew Maurer, MD, Arnold and Arlene Professor of Cardiology at Columbia University, explained the standard of care and current management tactics for individuals with hATTR Amyloidosis.

This morning, it was announced that Invitae Corporation and Sarepta Therapeutics will be expanding its partnership to continue assisting clinicians in identifying patients with Duchenne muscular dystrophy (DMD).

PLENVU, a one-liter solution from Salix Pharmaceuticals, is expected to reach US markets by the third quarter of 2018.

An analysis of 350,000-plus veterans found those who suffered concussions have at least a 2.51-fold increased risk of developing dementia.

The drug is approved for adults with depression, but exceptionally high placebo responses rendered its effects in children not statistically significant.

Antiretroviral drug levels were monitored to determine whether they are useful in guiding regimens for treatment-resistant HIV-AIDS.

Prenatal exposure to polycyclic aromatic hydrocarbons, as well as lifelong poverty, increased ADHD symptoms in children.

The Group Medical Director of Neuroscience at Genentech discussed the drug's background, which has evolved into continued multi-year trials.

The investigative vaccine from PaxVax is a virus-like particle currently undergoing phase 2B analysis for dosage rates.

The Advisor™ HD Grid Mapping Catheter, Sensor Enabled™ provides physicians with a detailed map of cardiac tissue to allow for more precise ablation therapy.

The U.S. FDA has granted Fast Track designation to PaxVax for development of its vaccine for prevention of the chkungunya virus.

FDA approves dual drug treatment, dabrafenib (Tafinlar) and trametinib (Mekinist), for Anaplastic Thyroid Cancer (ATC).

Rare Disease Report sat down with Brian Wamhoff, PhD, Head of Innovation at HemoShear Therapeutics, to discuss what the company is doing throughout the rare disease community and why it could have such an incredible impact in the near future.

According to new researche, a widely-used cholesterol-lowering drug increased a precursor of high-density lipoprotein (HDL) in models that mimicked liver cells of patients with Friedrich’s ataxia (FA).

An injectable soluble formulation of naturally-secreted growth factors developed by Histogen will begin its first company-sponsored clinical trial.

If FDA approved, it would be the first-in-class pharmacologic treatment for hyperbilirubinemia in newborns that contains a novel mechanism of action inhibiting bilirubin production at the source.

The U.S. FDA has approved coagulation factor Xa (recombinant), inactivated-zhzo (Andexxa) for patients treated with rivaoxaban and apixaban, when reversal of anticoagulation is necessary due to fatal or uncontrolled bleeding.

Andexanet alfa showed a median decrease in anti-Factor Xa activity from baseline of 97% for those on rivaroxaban and 92% for those on apixaban.

An assessment of the burden felt by caregivers of patients with cognitive impairment sought to identify a relationship between the level of burden and the level of impairment.

An initial high dose or high dose escalation can work best for Clostridium difficile treatment.

Dosing of the first patient in the Phase 2 clinical trial of AG10 in patients with ATTR cardiomyopathy, Eidos Therapeutics, Inc. has announced.

This is the first FDA-approved antidepressant for MDD to have data in US Prescribing Information showing a positive effect on processing speed.

All told, 68% of the group receiving a full dose of MDMA no longer qualified for a PTSD diagnosis compared to 29% in the active-placebo control group.

A massive genetic family migraine patient study found more defined risk scores for migraine subtypes.

On Monday, the FDA approved tisagenlecleucel (Kymriah) for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma who have relapsed or are ineligible for an autologous stem cell transplant (ASCT).

FusionVu allows for the fastest and simplest real-time targeting of prostate biopsies while also providing the highest resolution in real-time guidance for them, according to Exact Imaging.

Orchard Therapeutics announced that its gene therapy candidate, OTL-200, has been granted Rare Pediatric Disease designation for the treatment of metachromatic leukodystrophy.

Researchers examined the role of RPE dysfunction in patients with AMD and found this discovery demonstrated hUTC’s potential therapeutic value in the treatment of AMD.

The discovery could have researchers reconsidering treatment routes for pediatric patients.

The clinically relevant findings suggest that infant passive immunization with second-generation broad and potent bNAbs could be an effective strategy to block mother-to-child HIV transmission.