Rare diseases

LogicBio Therapeutics Announces SUNRISE Phase 1/2 Clinical Design for LB-001 for the Treatment of Methylmalonic Acidemia in Pediatric Patients

Monday, August 10, 2020 - 11:35am

Were pleased to announce our plans for developing LB-001 in pediatric patients with MMA.

Key Points: 
  • Were pleased to announce our plans for developing LB-001 in pediatric patients with MMA.
  • Early intervention in this vulnerable population is critical to combat the manifestation of irreversible clinical disease pathologies including neurological damage.
  • Six leading centers in the United States are expected to participate in the SUNRISE Phase 1/2 trial.
  • LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia.

FDA Approves Oral Treatment for Spinal Muscular Atrophy

Friday, August 7, 2020 - 7:26pm

This is the second drug and the first oral drug approved to treat this disease.

Key Points: 
  • This is the second drug and the first oral drug approved to treat this disease.
  • SMA is a hereditary disease that causes weakness and muscle wasting because patients lose lower motor neurons (nerve cells) that control movement.
  • The efficacy of Evrysdi for the treatment of patients with infantile-onset and later-onset SMA was evaluated in two clinical studies.
  • After 23 or more months of treatment, 81% of patients were alive without permanent ventilation, which is a noticeable improvement from typical disease progression without treatment.

Global Pompe Disease Market to 2030 - Insights, Epidemiology and Forecast - ResearchAndMarkets.com

Thursday, August 6, 2020 - 9:07am

The "Pompe Disease - Market Insights, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Pompe Disease - Market Insights, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • The report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of Pompe Disease (PD) from 2017 to 2030 segmented by seven major markets.
  • It covers the details of conventional and current medical therapies available in the Pompe Disease market for the treatment of the condition.
  • The Pompe Disease market report gives a thorough understanding of PD by including details such as disease definition, symptoms, causes, pathophysiology, and diagnosis.

Triplet Therapeutics To Present at Upcoming Events

Tuesday, August 4, 2020 - 1:08pm

ET;

Key Points: 
  • ET;
    William Blair BioTech Conference, Thursday, August 6, 2020, panel: 30 Years of RNA-Targeted Therapies: Where Are We Now?, at 2 p.m.
  • Triplet is initially focusing development of TTX-3360 in HD and may also evaluate it in other central nervous system indications such as spinocerebellar ataxias (SCAs), fragile X syndrome, and familial amyotrophic lateral sclerosis (ALS).
  • Triplet designs and develops potential therapeutics for REDs using its proprietary thRED Engine, which enables the Company to develop a single oligonucleotide targeting the DNA Damage Response (DDR) pathway to potentially treat multiple REDs.
  • Triplet is backed by investments from Atlas Venture, MPM Capital and Pfizer Ventures, along with Invus, Partners Innovation Fund and Alexandria Venture Investments.

Cerecor Receives Rare Pediatric Disease Designation for CERC-006 in Lymphatic Malformations

Tuesday, August 4, 2020 - 12:00pm

Lymphatic malformations are rare, non-malignant masses consisting of fluid-filled channels or spaces thought to be caused by the abnormal development of the lymphatic system.

Key Points: 
  • Lymphatic malformations are rare, non-malignant masses consisting of fluid-filled channels or spaces thought to be caused by the abnormal development of the lymphatic system.
  • It is estimated that 30,000 to 60,000 Americans have this condition in the United States.1
    We are very pleased to receive FDA Rare Pediatric Disease Designation for CERC-006.
  • The term rare disease or condition means any disease or conditions affecting less than 200,000 people in the United States.
  • Cerecor is a leading biopharmaceutical company focused on in development and commercialization of treatments for rare pediatric and orphan diseases.

Global Epidermolysis Bullosa Pipeline 2020: Drugs, Companies, Clinical Trials, R&D Updates, Status and Outlook - ResearchAndMarkets.com

Tuesday, August 4, 2020 - 11:47am

The "Epidermolysis Bullosa Pipeline Research Monitor, 2020 - Drugs, Companies, Clinical Trials, R&D Pipeline Updates, Status and Outlook" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Epidermolysis Bullosa Pipeline Research Monitor, 2020 - Drugs, Companies, Clinical Trials, R&D Pipeline Updates, Status and Outlook" report has been added to ResearchAndMarkets.com's offering.
  • Global Epidermolysis Bullosa market presents promising new product pipeline with NME Projects, pivotal trials, and rapidly phase-advancing therapeutic candidates.
  • This Epidermolysis Bullosa pipeline review explores high-potential early to late-stage pipeline projects with a continued focus on new insights, accelerated processes, and pipeline progression.
  • The competitive intelligence report on Epidermolysis Bullosa presents detailed insights into therapeutic drug pipeline development, industry news, deals, and analysis across the length and breadth of the Epidermolysis Bullosa pipeline.

Global Paroxysmal Nocturnal Hemoglobinuria Pipeline Monitor, 2020 - ResearchAndMarkets.com

Tuesday, August 4, 2020 - 10:52am

The "Paroxysmal Nocturnal Hemoglobinuria Pipeline Research Monitor, 2020 - Drugs, Companies, Clinical Trials, R&D Pipeline Updates, Status and Outlook" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Paroxysmal Nocturnal Hemoglobinuria Pipeline Research Monitor, 2020 - Drugs, Companies, Clinical Trials, R&D Pipeline Updates, Status and Outlook" report has been added to ResearchAndMarkets.com's offering.
  • Global Paroxysmal Nocturnal Hemoglobinuria market presents promising new product pipeline with NME Projects, pivotal trials, and rapidly phase-advancing therapeutic candidates.
  • This Paroxysmal Nocturnal Hemoglobinuria pipeline review explores high-potential early to late-stage pipeline projects with a continued focus on new insights, accelerated processes, and pipeline progression.
  • The competitive intelligence report on Paroxysmal Nocturnal Hemoglobinuria presents detailed insights into therapeutic drug pipeline development, industry news, deals, and analysis across the length and breadth of the Paroxysmal Nocturnal Hemoglobinuria pipeline.

FDA Approves EPIDIOLEX® (cannabidiol) Oral Solution to Treat Seizures Associated with Tuberous Sclerosis Complex

Monday, August 3, 2020 - 12:00pm

FDA approval of this new indication is exciting news for those with refractory seizures due to tuberous sclerosis complex, said Justin Gover, GWs Chief Executive Officer.

Key Points: 
  • FDA approval of this new indication is exciting news for those with refractory seizures due to tuberous sclerosis complex, said Justin Gover, GWs Chief Executive Officer.
  • FDA approval of EPIDIOLEX in TSC is a tremendous step forward and our community applauds this positive development, said Kari Luther Rosbeck, President and CEO of the Tuberous Sclerosis Alliance.
  • In the U.S., EPIDIOLEX is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome or tuberous sclerosis complex (TSC) in patients one year of age and older.
  • EPIDIOLEX (cannabidiol) oral solution is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC) in patients 1 year of age and older.

Tuberous Sclerosis Alliance Lauds FDA Approval of Epidiolex® to Treat Seizures in People with Tuberous Sclerosis Complex

Saturday, August 1, 2020 - 2:43am

SILVER SPRING, Md., July 31, 2020 /PRNewswire/ --Today, the Tuberous Sclerosis Alliance (TS Alliance) lauds the U.S. Food and Drug Administration's (FDA's) approval of Epidiolex oral solution to treat seizures in people one year of age and older who have tuberous sclerosis complex (TSC).

Key Points: 
  • SILVER SPRING, Md., July 31, 2020 /PRNewswire/ --Today, the Tuberous Sclerosis Alliance (TS Alliance) lauds the U.S. Food and Drug Administration's (FDA's) approval of Epidiolex oral solution to treat seizures in people one year of age and older who have tuberous sclerosis complex (TSC).
  • "Up to 85 percent of those affected by TSC experience seizures at some point in their lifetime," explained TS Alliance President & CEO Kari Luther Rosbeck.
  • In 2018, the FDA approved the drug to treat people two years of age and older with Lennox-Gastaut syndrome (LGS) or Dravet syndrome.
  • Established in 1974, the Tuberous Sclerosis Alliance is an internationally recognized nonprofit organization dedicated to finding a cure for tuberous sclerosis complex while improving the lives of those affected.

Fortress Biotech Announces Positive Opinion on Orphan Drug Designation Received from the European Medicines Agency for CUTX-101, Copper Histidinate, for the Treatment of Menkes Disease

Friday, July 31, 2020 - 12:30pm

The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug, Fast Track and Rare Pediatric Disease Designations to CUTX-101 for the treatment of Menkes disease.

Key Points: 
  • The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug, Fast Track and Rare Pediatric Disease Designations to CUTX-101 for the treatment of Menkes disease.
  • Orphan Drug Designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products.
  • CambPharma Solutions (CY) Limited submitted the Orphan Drug Designation application on behalf of Cyprium, as its agent in the EU.
  • Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of copper transporter ATP7A.