Neurological disorders

Akcea and Ionis Announce Publication of Long-Term Clinical Data of TEGSEDI® in Patients with Polyneuropathy Driven by Hereditary Transthyretin Amyloidosis Demonstrating Sustained Improvements and Even Greater Stabilization in Patients Starting Earlier Tr

Thursday, May 28, 2020 - 12:00pm

TEGSEDI is a once-weekly, at-home subcutaneous injection that targets the polyneuropathy of hATTR amyloidosis at its source by reducing production of the TTR protein.

Key Points: 
  • TEGSEDI is a once-weekly, at-home subcutaneous injection that targets the polyneuropathy of hATTR amyloidosis at its source by reducing production of the TTR protein.
  • Among those who participated in the ongoing OLE study, 85 continued to receive TEGSEDI and 50 switched from placebo to TEGSEDI.
  • There was also no evidence of increased risk for grade 4 thrombocytopenia or acute glomerulonephritis with increased duration of exposure to TEGSEDI.
  • TEGSEDI also received marketing authorization in theEuropean Unionand Canada for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis.

Impact of Baseline Dyskinesia on Safety and Efficacy of NOURIANZ® (istradefylline) in Patients with Parkinson's Disease Presented During "Virtual" European Academy of Neurology Meeting

Tuesday, May 26, 2020 - 10:15pm

These findings were presented during the 6th Congress of the European Academy of Neurology (EAN).

Key Points: 
  • These findings were presented during the 6th Congress of the European Academy of Neurology (EAN).
  • "The results being presented at EAN suggest that dyskinesia is observed more often in patients with baseline dyskinesia before istradefylline was added to the treatment regimen and that the overall efficacy of istradefylline was not affected by patients' baseline status.
  • While these data provide further information, physicians should continue to monitor patients for dyskinesia or exacerbation of existing dyskinesia during NOURIANZ treatment.
  • Impact of Baseline Dyskinesia on the Safety and Efficacy of Istradefylline, an Adenosine A2A Receptor Antagonist, in Patients with Parkinson's Disease: a Pooled Analysis of 8 Clinical Studies.

CytRx Corporation Highlights Orphazyme's Receipt of FDA Fast Track Designation for the Development of Arimoclomol in Amyotrophic Lateral Sclerosis (ALS)

Tuesday, May 26, 2020 - 2:00pm

Fast Track is a designation by the FDA of an investigational drug for expedited review to facilitate development of drugs which treat a serious or life-threatening condition and fill an unmet medical need.

Key Points: 
  • Fast Track is a designation by the FDA of an investigational drug for expedited review to facilitate development of drugs which treat a serious or life-threatening condition and fill an unmet medical need.
  • We are very pleased that the Company has received Fast Track Designation," said Steven A. Kriegsman, CytRx's Chairman and CEO.
  • Orphazyme is testing arimoclomol in four indications including amyotrophic lateral sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM).
  • Arimoclomol, the company's lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis.

Ipsen: New Surveys Show Over 80% of Patients with Spasticity and Cervical Dystonia Treated with Botulinum Toxin-A Experience Debilitating Symptom Recurrence

Saturday, May 23, 2020 - 6:00am

The surveys involved over 400 respondents from five countries, living with spasticity or cervical dystonia and receiving botulinum neurotoxin type A (BoNT-A) injections.

Key Points: 
  • The surveys involved over 400 respondents from five countries, living with spasticity or cervical dystonia and receiving botulinum neurotoxin type A (BoNT-A) injections.
  • Symptom recurrence significantly impacted patients quality of life, including sleep, relationships, performance of daily tasks and working lives.
  • Eligible participants were over 18 years old and had (or cared for someone with) spasticity or cervical dystonia (CD) treated with BoNT-A for at least one year.
  • How do patients with cervical dystonia (CD) experience their botulinum neurotoxin type a (BoNT-A) treatment cycle: results from an international online survey.

NeurologyLive® Adds Cure SMA to Its Strategic Alliance Partnership Program

Tuesday, May 19, 2020 - 7:03pm

NeurologyLive, a multimedia platform dedicated to providing health care professionals who treat neurological diseases with direct access to expert-driven, practice-changing news and insights in neurology, announces that Cure SMA is now part of its Strategic Alliance Partnership (SAP) program.

Key Points: 
  • NeurologyLive, a multimedia platform dedicated to providing health care professionals who treat neurological diseases with direct access to expert-driven, practice-changing news and insights in neurology, announces that Cure SMA is now part of its Strategic Alliance Partnership (SAP) program.
  • Cure SMA is the leading national organization dedicated to finding effective treatments and, ultimately, a cure for spinal muscular atrophy, or SMA.
  • Cure SMA does amazing work driving research and supporting the SMA community, said Mike Hennessy Jr., president and CEO of MJH Life Sciences, parent company of NeurologyLive.
  • Cure SMA is striving to create a world without SMA, one of the leading genetic causes of death for infants.

Blood-Brain Barrier Markets Insights and Forecast to 2030 by Type of Molecule, Target Disease Indications, Key Contributing Technologies, Leading Drug Developers and Key Geographical Regions

Tuesday, May 19, 2020 - 3:45pm

Parkinson's disease and stroke, are characterized by a significant unmet need for effective treatment options.

Key Points: 
  • Parkinson's disease and stroke, are characterized by a significant unmet need for effective treatment options.
  • In 2019, it was reported that nearly 1.5 billion people suffer from some form of disorder affecting the CNS across the globe.
  • In fact, the incidence of CNS disorders is anticipated to increase by 12% by 2030.
  • In 2019, nearly 5.8 million Americans were reported to have been diagnosed with Alzheimer's disease.

New Video from PANDAS Network Shows How Infections Attack Brains and Result in Devastating PANDAS/PANS Symptoms

Monday, May 18, 2020 - 8:30pm

The conditions are caused by an autoimmune reaction triggered by infections such as strep throat, walking pneumonia, viruses and Lyme disease, which result in inflammation of the child's brain.

Key Points: 
  • The conditions are caused by an autoimmune reaction triggered by infections such as strep throat, walking pneumonia, viruses and Lyme disease, which result in inflammation of the child's brain.
  • That attack causes neuroinflammation and neuronal dysfunction, leading to the abrupt onset of severe neurological and/or psychiatric symptoms.
  • "The loss of taste and smell in BGE patients was an unexpected clue," said Diana Pohlman, director of the nonprofit PANDAS Network .
  • PANDAS Network is dedicated to improving the diagnosis and treatment of children with PANDAS (Pediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections) and PANS (Pediatric Acute-onset Neuropsychiatric Syndrome).

China Approves AUSTEDO® For Treating Chorea Associated with Huntington's Disease and Tardive Dyskinesia in Adults

Monday, May 18, 2020 - 12:00pm

This is the second approval and expected launch in China of a Teva specialty medicine, following the recent launch of TREANDA.

Key Points: 
  • This is the second approval and expected launch in China of a Teva specialty medicine, following the recent launch of TREANDA.
  • The approval of AUSTEDO in China is an exciting milestone for Teva," said Gianfranco Nazzi, Executive Vice President, International Markets, at Teva.
  • Tardive dyskinesia is a movement disorder characterized by repetitive and uncontrollable movements of the tongue, lips, face, trunk and extremities.
  • AUSTEDO is a vesicular monoamine transporter 2 (VMAT2) inhibitor approved by the U.S. Food and Drug Administration for the treatment of tardive dyskinesia in adults and for the treatment of chorea associated with Huntingtons disease.

Final results from the ProCID study of efficacy and safety of 3 different dosages of Panzyga® (NewGam) in patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) presented at AAN 2020

Monday, May 18, 2020 - 9:55am

ProCID was the first prospective study to compare different maintenance doses of Panzyga (NewGam), an intravenous immunoglobulin (IVIg) in patients with chronic inflammatory demyelinating polyneuropathy (CIDP).

Key Points: 
  • ProCID was the first prospective study to compare different maintenance doses of Panzyga (NewGam), an intravenous immunoglobulin (IVIg) in patients with chronic inflammatory demyelinating polyneuropathy (CIDP).
  • The results of the study confirmed the efficacy of Panzyga in adults with CIDP at the standard dose of 1.0 g/kg every 3 weeks.
  • The results of the ProCID study confirm the efficacy and safety of Panzyga in patients with CIDP and suggest that the standard dosing regimen may not be optimal in some patients.
  • Chronic inflammatory demyelinating polyneuropathy disease activity status, Recommendations for clinical research standards and use in clinical practice.

AC Immune CEO to Showcase Novel Target Programs at UBS Global Healthcare Conference

Friday, May 15, 2020 - 12:00pm

LAUSANNE, Switzerland, May 15, 2020 (GLOBE NEWSWIRE) -- AC Immune SA (NASDAQ: ACIU), a Swiss-based, clinical-stage biopharmaceutical company with a broad pipeline focused on neurodegenerative diseases, today announced that it will showcase key programs focused on novel targets in neurodegenerative diseases at next weeks UBS Virtual Global Healthcare Conference (May 1820, 2020).

Key Points: 
  • LAUSANNE, Switzerland, May 15, 2020 (GLOBE NEWSWIRE) -- AC Immune SA (NASDAQ: ACIU), a Swiss-based, clinical-stage biopharmaceutical company with a broad pipeline focused on neurodegenerative diseases, today announced that it will showcase key programs focused on novel targets in neurodegenerative diseases at next weeks UBS Virtual Global Healthcare Conference (May 1820, 2020).
  • Speaking on May 20th, Prof. Andrea Pfeifer, Ph.D., CEO of AC Immune SA, will outline the cutting-edge science behind AC Immunes therapeutic and diagnostic programs in TDP-43 and alpha-synuclein which are amongst the most comprehensive in the field.
  • This highlights the need for the precision medicine approach pioneered byAC Immune to achieve earlier and more accurate diagnosis.
  • AC Immune SA is a Nasdaq-listed clinical-stage biopharmaceutical company, which aims to become a global leader in precision medicine for neurodegenerative diseases.