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TAG: Rare Disease

Jul 27, 2023

Congressman Bobby Scott Tours ReAlta Life Sciences’ Biotech Research Facility, Highlights Importance of Rare Disease Innovation

Apr 5, 2023

Global Rare Disease Therapeutics Market Report 2023: Players Include AbbVie, Novo Nordisk, Sanofi, Bayer, Amgen and Eisai - ResearchAndMarkets.com

Mar 31, 2023

Scribe Therapeutics to Participate in Guggenheim Healthcare Talks: Genomic Medicines and Rare Disease Days

Mar 29, 2023

Epic Bio to Participate in the Guggenheim Healthcare Talks - Genomic Medicines and Rare Disease Days

Mar 27, 2023

ReCode Therapeutics to Present at Guggenheim Healthcare Talks: Genomic Medicines and Rare Disease Days

Dec 5, 2022

APR Applied Pharma Research (una controllata di Relief Therapeutics) è tra i finalisti del Rare Disease International Film Festival 2022 English

Nov 21, 2022

APR Applied Pharma Research (a Subsidiary of Relief Therapeutics) is a Finalist in the 2022 Rare Disease International Film Festival USA - English France - Français España - español USA - English USA - English Deutschland - Deutsch Italia - Italiano Italia - Italiano

Nov 21, 2022

APR Applied Pharma Research (a Subsidiary of Relief Therapeutics) is a Finalist in the 2022 Rare Disease International Film Festival USA - English USA - Français USA - español USA - English USA - Deutsch USA - Italiano

Aug 17, 2022

Kriya Appoints Pedro Huertas M.D., Ph.D., as Chief Medical Officer of its Rare Disease Division

Jun 28, 2022

Advicenne Announces Commercialization Agreement with Taïba Healthcare for Sibnayal in Middle-East

May 19, 2022

Be Bio and Resilience Announce Strategic Collaboration to Manufacture Engineered B Cells, a New Class of Cellular Medicines

Apr 14, 2022

Be Bio Closes $130 Million Financing to Pioneer Engineered B Cells, a New Class of Cellular Medicines

Mar 10, 2022

Scenic Biotech Announces $31 Million Financing to Progress Pipeline of Genetic Modifiers in Cancer and Rare Diseases

Feb 16, 2022

Star Therapeutics emerges from stealth mode raising $100M in financing since inception to be an innovation engine for rare disease therapies

Jan 7, 2022

Kriya Expands Gene Therapy Pipeline and Establishes Its Rare Disease Therapeutic Area Division With the Acquisition of Warden Bio

Dec 14, 2021

HemoShear Therapeutics Appoints Patrick Horn, MD, PhD, Chief Medical Officer

Nov 1, 2021

Aceragen Announces Acquisition of Arrevus, Expands Late-Stage Rare Disease Pipeline

Oct 5, 2021

OpenBench and HemoShear Therapeutics Agree to Pursue Rare Disease Target Using Artificial Intelligence

Sep 13, 2021

Cycle Pharmaceuticals Launches Cycle Vita™, a Dedicated Support Platform for Rare Disease Patients

Aug 31, 2021

VectivBio to Acquire Comet Therapeutics and Host RandD Day

Aug 24, 2021

Shape Therapeutics enters into a strategic research collaboration with Roche to advance breakthrough AAV-based RNA editing technology for neuroscience and rare disease indications

Jul 27, 2021

RareStone Inc. Announces Strategic Collaboration with Tencent on a Service Ecosystem Focused on Rare Disease Patients in China

May 5, 2021

Rare Disease Therapeutics, Inc. Announces New Expanded FDA Approved Indication for Antivenom ANAVIP for Treatment of North American Pit Viper Envenomation

Apr 19, 2021

Amylyx Pharmaceuticals Appoints Chris Aiello to Head of Canada and General Manager

Apr 19, 2021

Citrine Medicine and Sinopharm Group Announce Strategic Partnership to Broaden and Accelerate Access to Rare Disease Drugs in China

Apr 12, 2021

Mirum Pharmaceuticals and Vivet Therapeutics Enter into Exclusive Worldwide Option and License Agreement for Vivet’s Gene Therapy Programs Targeting Progressive Familial Intrahepatic Cholestasis

Mar 22, 2021

Citrine Medicine Announces Receipt of Clinical Trial Waiver from the National Medical Products Administration (NMPA) in China for Narcolepsy Therapy Wakix (pitolisant) and Partnership With Chinese Alliance for Rare Diseases (CARD)

Mar 2, 2021

Rallybio Reveals Details of Two Pipeline Programs with the Potential to Transform the Lives of Patients with Rare Diseases

Feb 26, 2021

Dynacure Recognizes Rare Disease Day 2021

Feb 25, 2021

M6P Therapeutics Supports Rare Disease Day 2021 and Joins Global Movement to Increase Awareness of Rare Diseases

Feb 11, 2021

Ensoma Announces Strategic Collaboration with Takeda to Accelerate Next-Generation In Vivo Gene Therapies

Feb 10, 2021

HemoShear Therapeutics Raises $40 Million in Series A Financing to Advance Rare Disease Portfolio

Oct 28, 2020

Citrine Medicine Advances Efforts to Create the Very First Rare Disease Ecosystem in China Through Strategic Partnership with Bioprojet to License Exclusive Rights for the Use of Orphan Narcolepsy Therapy Wakix (pitolisant)

Sep 7, 2020

Cycle Pharmaceuticals Secures $25 Million Debt Financing

Aug 25, 2020

Tammy Sarnelli Joins Amylyx Pharmaceuticals as Global Head of Regulatory Affairs

Jul 21, 2020

Forge Biologics Launches with $40 Million Series A Financing to Manufacture and Develop Gene Therapies

May 19, 2020

Rallybio Completes $145 Million Series B Financing and Announces Plans to Advance Lead Product Candidate into the Clinic in 2H 2020

Mar 26, 2020

Evox Therapeutics and Takeda Sign Multi-target Rare Disease Collaboration

Dec 9, 2019

Healx Joins Forces with Boehringer Ingelheim to Discover New Treatment Approaches for Rare Neurological Diseases

Sep 25, 2019

HemoShear Therapeutics Appoints Brian A. Johns, PhD, Chief Scientific Officer

Aug 8, 2019

Dynacure Receives Orphan Drug Designation from the US FDA for DYN101, an Antisense Medicine to Treat Rare Disease 'Centronuclear Myopathies'

May 8, 2019

Pfizer Acquires Clinical-Stage Biotech Therachon

Apr 30, 2019

Dynacure Announces Approval of Clinical Trial Application for DYN101, an Antisense Medicine to Treat Rare Disease 'Centronuclear Myopathies'

Mar 12, 2019

Tiburio Therapeutics Inc. Appoints Nerissa Kreher, MD, Chief Medical Officer

Jan 23, 2019

CAMP4 Therapeutics Announces Rare Disease Research Collaboration with Alnylam

Jan 9, 2019

EUSA Pharma Completes Acquisition of Global Rights to SYLVANT (Siltuximab) and Presents Company Update at 37th J.P. Morgan Healthcare Conference

Jan 8, 2019

CANbridge Pharmaceuticals and WuXi Biologics Expand Rare Disease Therapeutics Partnership

Dec 20, 2018

Precision BioSciences Appoints Rare Disease Executive Shalini Sharp to Its Board of Directors

Nov 8, 2018

Ascendis Pharma Announces Formation of VISEN Pharmaceuticals to Develop and Commercialize TransCon™ Endocrinology Rare Disease Therapies in China

Oct 23, 2018

CANbridge Pharmaceutical and WuXi Biologics Enter into Strategic Partnership for Rare Disease Therapeutics

Oct 23, 2018

Himalaya Therapeutics Announces Appointment of Nicholas Desjardins as Chief Financial Officer

Sep 5, 2018

Palvella Therapeutics Appoints Rare Disease Senior Executive Elaine J. Heron, PhD to Board of Directors

Aug 22, 2018

Jupiter Orphan Therapeutics Announces Favorable Data Which Expands JOTROL's Applications to the Estimated $5 Billion Mitochondrial Rare Disease Market

Aug 14, 2018

Sigilon Therapeutics Announces Leadership Transition and Appoints Rare Disease Pioneer Rogerio Vivaldi, M.D., as Chief Executive Officer

Aug 9, 2018

Therachon Raises $60 Million Mezzanine Financing to Advance Lead Program in Achondroplasia and to Expand Rare Disease Pipeline

May 17, 2018

Stoke Therapeutics Expands Leadership Team, Building on Deep Expertise in Oligonucleotide Chemistry and Rare Disease Research and Development

Feb 28, 2018

Spruce Biosciences Launches Natural History Study for Congenital Adrenal Hyperplasia in Time for World Rare Disease Day

Jan 4, 2018

Stoke Therapeutics Announces $40 Million Series A Financing to Create Pioneering New Medicines that Restore Gene Expression in Severe Genetic Diseases

Dec 13, 2017

Aprecia Pharmaceuticals and Cycle Pharmaceuticals Partner to Develop 3D-Printed Orphan Drugs

Oct 27, 2017

BERG Honored with Partner in Progress Award from DEBRA of America at Annual Benefit for Rare Disease Epidermolysis Bullosa (EB)

Oct 20, 2017

Agilis Biotherapeutics Announces Presentations on Angelman Syndrome Program at ESGCT and NORD Rare Disease Summit

Oct 17, 2017

Cydan Raises $34 Million to Accelerate New Treatments for Rare Genetic Diseases

May 3, 2017

Cydan Development Announces the Acquisition of Its First Rare Disease Spin-out, Vtesse, Inc. by Sucampo Pharmaceuticals, Inc.

Feb 28, 2017

Spyryx Biosciences Marks Rare Disease Day With an Update on Clinical Trials for SPX-101

Feb 28, 2017

Therachon Supports Rare Disease Day® and Joins Global Movement to Raise Important Awareness for Rare Diseases

Feb 28, 2017

AMO Pharma Joins with Leaders in Patient and Caregiver Advocacy to Commemorate Rare Disease Day

Feb 28, 2017

Cydan Development Celebrates Rare Disease Day with Gift to Support the Children's National Rare Disease Institute

Feb 28, 2017

Therachon Supports Rare Disease Day and Joins Global Movement to Raise Important Awareness for Rare Diseases

Feb 6, 2017

Aura Biosciences Receives FDA Clearance of Investigational New Drug Application for Light-activated AU-011 for the Treatment of Ocular Melanoma

Feb 29, 2016

Cydan Development Celebrates Rare Disease Day with Gift to Support the NORD Natural History Project

Jun 9, 2015

AM-Pharma Receives FDA and EMA Orphan Drug Designation to Treat Hypophosphatasia

Feb 26, 2015

CORRECTING and REPLACING Recursion Pharmaceuticals Wins $1.5M NIH Grant for Rare Disease Drug Discovery

Feb 23, 2015

Orphan Drug Accelerator Cydan Supports Rare Disease Day and Joins Patients, Advocates and Massachusetts Legislators to Raise Awareness for Rare Diseases

Jan 7, 2015

Leading Life Science Syndicate Commits $25 Million to Series A Funding to Launch Vtesse, Inc., the First Rare Disease Company Spun Out of Cydan Development, Inc. Deutschland - Deutsch France - Français USA - English España - español

Dec 2, 2014

Preclinical Data Published on AM-Pharma’s recAP to Treat Ultra Rare Disease Hypophosphatasia

Feb 28, 2013

Rare disease day: Boehringer Ingelheim announces initiation of pivotal trial investigating volasertib* in patients with acute myeloid leukaemia