Rare Disease Articles & Analysis

PHILADELPHIA, Aug. 04, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced financial results for the quarter ended June 30, 2022. Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “Through the ...

By Amicus Therapeutics, Inc.

Holdings Limited (Tencent),based in Shenzhen,China,signed a strategic collaboration memorandum to develop a service ecosystem focused on rare disease patients in China. The combined effort will promote education of rare disease,focus social awareness on rare disease,and improve accessibility of medical information and services for rare disease by working together to provide innovative digital ...

By RareStone Group

As several gene therapy approvals for rare disease treatment gain steam in 2023, a comprehensive set of gene therapy development solutions is released to pave richer avenues for rare disease research. This path-breaking move sees the integration of cutting-edge technology with state-of-art research facilities to amplify the possibilities of devising effective remedies for rare diseases. Rare ...

By Protheragen

Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, announced that John McKew, PhD, President and Chief Scientific Officer of Lumos Pharma, will participate in a panel discussion at the Cantor Rare Disease Symposium at the end of March 2022. Event: Cantor Rare Disease Symposium – March 28-30 Presentation: Panel ...

By Lumos Pharma

Pioneering technology platform rapidly uncovers the genetic causes of unsolved diseases and redefines the understanding of rare and complex genetic diseases. Melville, NY, (February 28, 2017) Population Bio (PB) – a global leader in gene discovery announced, in support of World Rare Disease Day, its continued investment in its CNV Beacon® gene discovery platform which delivers new ...

By Population Bio, Inc. a Precision Medicine Company

Protheragen, a dedicated preclinical research service provider specializing in rare diseases, announces its comprehensive diagnostic development services aimed at empowering researchers in the realm of rare disease research and drug development. These diagnostic services are designed to support the scientific community in identifying, understanding, and ultimately treating rare diseases that ...

By Protheragen

Haymarket Medical Network today launched Rare Disease Advisor, a new digital resource for healthcare professionals (HCPs) to help them recognize, diagnose, refer and treat rare diseases. RareDiseaseAdvisor.com fills an information gap for HCPs by providing difficult to find information on rare diseases in one convenient place, enabling physicians and care teams with a comprehensive digital ...

By Haymarket Media Group

3DBio Therapeutics, an emerging leader in the field of 3D-bioprinting for human therapeutic use, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation for AuriNovo™, an investigational combination product for reconstruction of the outer ear in patients with microtia. Microtia is a rare congenital disorder causing the outer ear ...

By 3DBio Therapeutics

Healx, the AI-powered, patient-inspired technology company pioneering the next generation of drug discovery, today announced an exciting partnership with Ono Pharmaceutical Co., Ltd., one of Japan’s largest pharmaceutical companies and a global leader in the field of R&D-orientated drug discovery. The collaboration will leverage Healx’s innovative indication expansion technology ...

By Healx Ltd.

Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, announced that the Company will present at the H.C. Wainwright Virtual BIOCONNECT Conference being held January 10-13, 2022. A webcast of the presentation will be available at 7:00AM ET on January 10th through the following link: ...

By Lumos Pharma

Capricor Therapeutics (NASDAQ: CAPR) a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, announced today that Dr. Linda Marbán, CEO of Capricor, will participate in a panel discussion at the virtual Cantor Fitzgerald Rare Orphan Disease Summit on March 30, 2022. Please see ...

By Capricor Therapeutics, Inc.

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) and Deep Genomics today announced that the companies have entered into a preclinical collaboration that will use Deep Genomics’ artificial intelligence drug discovery platform (The AI Workbench) to identify oligonucleotide drug candidates in four rare disease indications with high unmet need. Deep Genomics will receive an undisclosed upfront ...

By Deep Genomics

HemoShear Therapeutics, Inc., a privately held clinical stage biotechnology company, today announced that it will participate in SVB Leerink Biopharma Private Company Connect being held virtually October 26-27. Investor one-on-one meetings with the HemoShear management team may be requested through SVB Securities. About HemoShear Therapeutics HemoShear Therapeutics, Inc. is a privately held ...

By HemoShear Therapeutics, Inc.

The narcolepsy drug Wakix can now be prescribed to patients in China with urgent unmet medical needs BOSTON, Mass., and Boao, Hainan, China, May 07, 2021 – Citrine Medicine, a China-based rare disease therapeutics company, today announced that the narcolepsy therapy Wakix® (pitolisant) has been approved by the Health Commission and Medical Products Administration of Hainan Province ...

By RareStone Group

HemoShear Therapeutics, Inc., a privately held clinical stage biotechnology company, today announced that Brain Wamhoff, Interim CEO, will present at the 34th Annual Piper Sandler Healthcare Conference being held November 29 – December 1 in New York City. The HemoShear management team will also participate in one-on-one meetings with investors. Meetings may be requested through Piper ...

By HemoShear Therapeutics, Inc.

Infant Bacterial Therapeutics AB (publ) publishes today the Annual Report for 2021. The report is available on IBT’s website ibtherapeutics.com under the section “Investors & Media – Financial Reports” and is available in printed form for those that have requested. A copy can be requested by contacting IBT’s office via info@ibtherapeutics.com. About Infant ...

By Infant Bacterial Therapeutics AB

Experienced machine learning and engineering leader Andrew Watson joins Healx from technology giant Dyson as VP of Artificial Intelligence. In his new role, Andrew will deepen the investment in Healx’s artificial intelligence team and scale Healx’s technical capabilities in order to identify and progress more novel treatments for rare diseases. Andrew’s appointment reinforces ...

By Healx Ltd.

LISCure Biosciences Inc., a leading biotech focused on microbial-based therapeutics, announced that it has executed a research collaboration agreement with Mayo Clinic for new drug development for rare liver diseases. LISCure has been conducting joint research with Mayo Clinic for NASH drugs since 2021, and this new collaboration is an agreement signed under the leadership of Nicholas F. ...

By LISCure Biosciences Inc.

VEO Ophthalmics, LLC announced today that, in honor of Rare Disease Day, it is collaborating with the Masket Foundation to raise awareness of aniridia and treatment options for aniridia. Aniridia is a serious ophthalmic rare disease in which a patient’s iris does not develop properly. VEO Ophthalmics is the U.S. distributor for the CUSTOMFLEX® ARTIFICIALIRIS, a custom-made artificial ...

By VEO Ophthalmics, LLC

RareStone to seek marketing authorization for IMCIVREE to treat obesity due to biallelic POMC, PCSK1 and LEPR deficiencies and Bardet-Biedl and Alström syndromes in mainland China, Hong Kong and Macau – BOSTON, December 2, 2021 -- RareStone Group, a rare disease focused company aiming to establish the first rare disease ecosystem in China, Rhythm Pharmaceuticals, Inc. (Nasdaq: ...

By RareStone Group