Progressive Supranuclear Palsy Treatment, ASN120290, Gets Orphan Drug Designation
The FDA has granted an orphan drug designation to Asceneuron SA for ASN120290, for the treatment of progressive supranuclear palsy.
Mutations in TRAF7 Gene Associated with Mysterious Multisystem Disorder
Researchers at Baylor College of Medicine find that mutations in the gene
are associated with a multisystem disorder presented by 7 patients.
Amyotrophic Lateral Sclerosis: Exploring the Current & Future Treatment Landscape
More treatments are needed for ALS, beyond the available riluzole and edaravone.
Top 5 Rare Disease News of the Week—July 8, 2018
Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.
Phase 3 Trial Evaluating ZX008 for Dravet Syndrome Meets Primary Endpoint
The phase 3 trial evaluating ZX008 as a treatment for Dravet syndrome met its primary endpoint.
FDA Grants Orphan Drug Designation to ACE-083 for FSHD Treatment
The FDA has granted orphan drug designation to Acceleron Pharma Inc for its product, ACE-083, for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
Brineura Brings Hope as Batten Patients Await Additional Therapies
The FDA’s approval of Brineura in April 2017 was a landmark moment for patients with the CLN2 form of the disease, but those with the other 13 forms of Batten are still waiting.
Friedreichs Ataxia Treatment, Omaveloxolone, Receives EU Orphan Drug Designation
Friedreich’s ataxia treatment, omaveloxolone, received orphan drug designation from the European Commission.
D-PUFA Drug Candidate Shows Cognitive Improvement in Huntington Disease Patients
An isotopic-reinforced polyunsaturated fatty acids shows a reduction in cognitive deficits in a preclinical
model of Huntington disease.
New Therapies on the Horizon for Huntington's Disease
Several promising new treatment developments are offering a ray of hope to Huntington’s patients, their at-risk children, and the clinicians who treat them.
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