Rare Partnering and Investor Forum – 9/14/2017 in Irvine, CA

June 26, 2017

Here’s all the info on our first ever Rare Disease Partnering Event! In this “Shark Tank” styled event we’ll welcome new ideas to help life and advance the rare disease community for all.

Click here to register for the event!

Drug Development

FDA approves Pharming’s Joenja for the treatment of APDS

Rare Daily Staff The U.S. Food and Drug Administration has approved Pharming Group’s Joenja for the treatment […]

Healthcare

NHS Patients with rare genetic disorders to be fast-tracked to earlier diagnosis and specialist care

Rare Daily Staff NHS England said it is launching a new service to fast track diagnosis and […]

Drug Development

European Commission Approves Amicus’ Pombiliti in Patients with Late-Onset Pompe Disease

Rare Daily Staff The European Commission has granted approval for Amicus Therapeutics’ Pombiliti, a long-term enzyme replacement […]

Drug Development

Ionis and AstraZeneca Report Positive Topline 66-Week Results of Eplontersen Phase 3 Study for Patients with ATTRv-PN

Rare Daily Staff Ionis Pharmaceuticals and AstraZeneca reported positive topline results from the 66-week analysis of the […]

Regulatory

NICE Recommends PTC’s AADC Gene Therapy

Rare Daily Staff The United Kingdom’s National Institute for Health and Care Excellence, the drug price watchdog, […]

Strategy

PTC and Innozyme CEOs Each Announce Their Retirement

Rare Daily Staff PTC Therapeutics Founding CEO Stuart Peltz said that he is stepping down after 25 […]

Diagnosis

2023 RARE Mental Health Grant Recipients Announced

Global Genes is pleased to announce the Rare Disease Patient Advocacy Organizations receiving 2023 RARE Mental Health […]

podcast

Seeking a Sustainable Business Developing on N-of-1 Therapies

The ability to pinpoint the underlying genetic causes of diseases and rapidly generate genetic medicines to address […]

Diagnosis

Global Genes Team Shares Their Stories on World Rare Disease Day

My daughter Juno was diagnosed with a very rare, severe neurodevelopmental condition called STXBP1-related disorder six years […]

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Rare Partnering and Investor Forum – 9/14/2017 in Irvine, CA

Related

FDA approves Pharming’s Joenja for the treatment of APDS

NHS Patients with rare genetic disorders to be fast-tracked to earlier diagnosis and specialist care

European Commission Approves Amicus’ Pombiliti in Patients with Late-Onset Pompe Disease

Ionis and AstraZeneca Report Positive Topline 66-Week Results of Eplontersen Phase 3 Study for Patients with ATTRv-PN

NICE Recommends PTC’s AADC Gene Therapy

PTC and Innozyme CEOs Each Announce Their Retirement

2023 RARE Mental Health Grant Recipients Announced

Seeking a Sustainable Business Developing on N-of-1 Therapies

Global Genes Team Shares Their Stories on World Rare Disease Day

Stay Connected