Remove 2018 Remove Clinical Development Remove Disease Remove Treatment
article thumbnail

New avenues for rare disease treatment

Drug Target Review

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. Most of these conditions are genetic in origin and the majority have no effective treatment.

Disease 113
article thumbnail

Taking a new approach to tackle neurodegenerative diseases

Drug Discovery World

William Erhardt , President, Head of Development & Operations of biotech Oligomerix, tells Lu Rahman about the company’s differentiated approach to developing disease-modifying therapeutics for neurodegenerative diseases. . With its recent award of $3.35 In the US alone, AD prevalence is expected to increase from 6.5

Disease 130
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Beyond immunotherapy: Could RNA modifying enzymes boost response rates? 

Drug Discovery World

The research underpinning the CTLA-4 and PD-1 technologies resulted in the Nobel Prize in Physiology or Medicine 2018 for the researchers and teams who pioneered them – James Allison of the University of California, Berkeley, and Tasuku Honjo at Kyoto University, respectively.

RNA 173
article thumbnail

Meet the Patient Advocate Panelists in Our Upcoming Webinar: Rare Disease Day 2023 Wrapped, What You Need to Take Away

Conversations in Drug Development Trends

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

Disease 52
article thumbnail

Three trends in the antibody-drug conjugate (ADC) market 

Drug Discovery World

According to NCBI, most of the ADCs developed so far are for use in the treatment of cancer, but there is plenty of potential for using ADCs to treat other diseases 1. To overcome this challenge, Synaffix has developed an innovative technology platform that can rapidly and efficiently convert any antibody into an ADC.

Marketing 245
article thumbnail

Roche’s ENSPRYNG approved by European Commission as first and only at-home subcutaneous treatment for neuromyelitis optica spectrum disorder (NMOSD)

The Pharma Data

ENSPRYNG is the first and only NMOSD treatment that is administered subcutaneously every four weeks, allowing home-dosing after appropriate training. Importantly, people with NMOSD now have the flexibility to administer treatment at home, which may alleviate the need to travel for hospital appointments.”.

article thumbnail

RYBREVANTTM (amivantamab-vmjw) in the Treatment of Patients with Advanced Non-Small.

The Pharma Data

The Janssen Pharmaceutical Companies of Johnson & Johnson today announced preliminary data from the Phase 1 CHRYSALIS study evaluating RYBREVANT TM (amivantamab-vmjw) for the treatment of patients with non-small cell lung cancer (NSCLC) with mesenchymal-epithelial transition (MET) exon 14 skipping (METex14) mutations. 4] , [5] , [6].