XTalks

NOVEMBER 10, 2023

Today, we are proud to further support the rare disease community by delivering Adzynma as the first FDA-approved therapeutic option for people with cTTP.” No adverse events were reported during the clinical trials following the administration of Adzynma. Adzynma’s approval is the second for Takeda this week.

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XTalks

NOVEMBER 7, 2022

Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials. Regulatory Approvals for CTC-Based Assays in Oncology.

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XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”

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XTalks

MARCH 10, 2023

This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher. Omaveloxolone Clinical Trials Skyclarys is an oral medication that contains omaveloxolone, an activator of the nuclear factor-erythroid 2 related factor 2 (Nrf2) pathway.

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XTalks

APRIL 11, 2024

After backing from the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) last month, Johnson and Johnson (J&J) and Bristol Myers Squibb (BMS) scored FDA approvals for the use of their B-cell maturation antigen (BCMA)-targeting CAR T-cell therapies as earlier line treatment options for multiple myeloma.

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XTalks

MAY 31, 2022

The precise cause of psoriasis is unknown, but risk factors include genetics and a family history of the condition, heavy alcohol consumption, stress, smoking and dry or cold weather. XTALKS WEBINAR: All Means All: The Road to Inclusivity in Clinical Trials. Clinical Trials of Vtama. Dermavant’s Future Targets.

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XTalks

NOVEMBER 27, 2023

Amicus Therapeutics has recently received approval from the US Food and Drug Administration (FDA) for its drugs Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).

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XTalks

DECEMBER 28, 2023

BillionToOne, a company specializing in molecular diagnostics, has partnered with Janssen Research & Development, LLC, a Johnson & Johnson division, to conduct the AZALEA Phase III clinical trial globally. The trial is set to occur across the US, the European Union (EU) and selected global locations.

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XTalks

MARCH 21, 2023

It is estimated that Rett syndrome affects 6,000 to 9,000 patients in the US, with a diagnosed population of approximately 4,500 patients, according to a press release from Acadia announcing Daybue’s approval. However, in animal studies, trofinetide has been shown to increase dendritic branching and synaptic plasticity signals.

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XTalks

NOVEMBER 30, 2023

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

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XTalks

MARCH 28, 2024

For CRC, treatment options that are targeting genetic mutations such as BRAF , KRAS and HER2 have been developed. Building Cancer Awareness and Empowering Métis Youth: Featuring Jordyn Playne, President, Métis Nation of Ontario Youth Council – Xtalks Life Science Podcast Ep.

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XTalks

FEBRUARY 9, 2024

Epilepsy has diverse etiologies, which are categorized into structural, genetic, infectious, metabolic, immune and unknown causes. Ztalmy marks the first FDA-approved treatment specifically designed for CDD and operates as a neuroactive steroid, acting as a positive allosteric modulator of the GABA A receptor.

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XTalks

JUNE 29, 2022

The new FDA approval gives Breyanzi (lisocabtagene maraleucel) the broadest patient eligibility of any CAR T-cell therapy in relapsed or refractory LBCL. Treatment with Breyanzi is a tailored method of treatment which involves the genetic reprogramming of T cells. Outcomes of Clinical Trials for Breyanzi.

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XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables.

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XTalks

SEPTEMBER 12, 2023

Life science podcasts have emerged as an invaluable tool for building connections with audiences in the digital era. Furthermore, we’ll explore the unique advertising opportunities that this platform offers, positioning brands at the forefront of the life science industry. The result?

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XTalks

AUGUST 21, 2023

The FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV) to Ipsen that can be used for subsequent drug applications that would not qualify for a priority review. The FDA approval of Sohonos is a breakthrough for the US FOP community,” said Howard Mayer, head of R&D at Ipsen, in the company’s news release.

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XTalks

FEBRUARY 28, 2022

Below are some facts and information about rare diseases, including rare disease clinical trials and orphan drugs. Are Most Rare Diseases Genetic? While 72 percent of rare diseases are genetic, not all are. Rare genetic diseases can be caused by either somatic or germline genetic changes.

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XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. In March, the US Food and Drug Administration (FDA) approved Daybue (trofinetide) , the first and only treatment for Rett syndrome. It affects around one in 10,000 girls born each year.

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XTalks

SEPTEMBER 22, 2022

On September 1, 2022, Boehringer Ingelheim Pharmaceuticals announced in a press release that the US Food and Drug Administration (FDA) approved Spevigo (spesolimab-sbzo) intravenous injections for GPP flares in adults. Spevigo is the first approved treatment option for GPP in adults. Clinical Trial Behind Spevigo.

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XTalks

MAY 1, 2023

Last week, the US Food and Drug Administration (FDA) granted accelerated approval to Biogen’s Qalsody (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 ( SOD1 ) gene.

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XTalks

JANUARY 26, 2024

By targeting and identifying specific genetic mutations, this technology could lead to the development of more precise diagnostic tools, enabling early intervention and treatment. By tailoring treatments based on an individual’s genetic makeup, it may allow for more effective and targeted therapies.

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XTalks

DECEMBER 4, 2023

The FDA said it is investigating the identified risk of developing the T-cell malignancies as secondary cancers with serious outcomes, including hospitalization and death, and also looking at the need for regulatory action. However, the FDA’s investigation has now revealed actual reports of T-cell malignancies linked to the therapies.

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XTalks

SEPTEMBER 17, 2021

The ALS Association is pushing for rapid FDA approval of Amylyx Pharmaceuticals Inc.’s Accelerated FDA Approval Modeled on Aduhelm. This has brought criticism that US regulators are moving too slow on a drug that has clinical promise and is being backed by other international regulators.

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XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies.

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XTalks

JUNE 28, 2023

The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial. DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness.

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XTalks

AUGUST 17, 2020

Sarepta Therapeutics’ Vyondys 53 (golodirsen) was approved in December of 2019, and along with the company’s first DMD drug — Exondys 51 (eteplirsen) — the company maintains they can now treat up to 20 percent of individuals with DMD in the US. Lurie Children’s Hospital of Chicago, said in a statement.

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XTalks

MARCH 25, 2021

Xtalks is proud to announce the launch of the Xtalks Life Science podcast. Subscribe to the Xtalks Life Science Podcast to never miss a new episode. Fresh Conversations About Life Science Topics. Sarah is passionate about science communication and journalistic integrity.

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XTalks

APRIL 5, 2024

Last month, Amylyx reported results from the global PHOENIX Phase III clinical trial that were unable to confirm the drug’s effectiveness. The life expectancy after symptoms develop is around two to five years.

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XTalks

FEBRUARY 5, 2024

In the dynamic landscape of oncology therapeutics, understanding the trends and performances of leading drugs is crucial for stakeholders across the healthcare and life sciences sectors. The top 40 best-selling oncology drugs in 2023, informed by 2022 sales statistics, mark significant milestones in cancer treatment and research.

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Pharmaceutical Technology

NOVEMBER 29, 2022

The company now plans to move this approach into a Phase III trial. PharmaTher also has an agreement in place with Case Western Reserve University to develop ketamine as a treatment for Rett syndrome, a rare genetic neurological disorder. In 1970, the FDA approved ketamine as an anesthetic.

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XTalks

MARCH 3, 2021

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.

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XTalks

DECEMBER 22, 2021

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. Here is a look at some life science trends to expect in the coming year, including key themes and topics in the pharmaceutical, biotechnology and medical device industries to keep an eye out for in 2022.

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XTalks

MAY 10, 2023

The exact cause of lupus is not entirely understood, but it is thought to be from a combination of genetic, environmental and hormonal factors. Lupkynis was the first FDA-approved oral medication specifically indicated for the treatment of LN. What Is the Theme of World Lupus Day 2023?

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XTalks

JUNE 15, 2022

If approved, NVX-CoV2373 would become the first available protein-based vaccine against COVID-19 in the US. Clinical Trial Results. These vaccines are highly effective, with clinical trial results showing approximately 95 percent effectiveness against the virus; the vaccines are also safe with mostly tolerable side effects.

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Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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XTalks

JUNE 8, 2021

In addition, recent data from a Phase I trial has shown ‘remarkable’ long-term results of the gene therapy in children with the disease. It was first approved in the US in 2019 to treat children below the age of two with SMA. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy?

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XTalks

APRIL 13, 2022

Pediatric rare disease clinical trials are vital to determining the safety and efficacy of medications for children living with a rare disease. About 80 percent of rare diseases have a genetic basis. Most rare diseases are chronic conditions, and many are clinically progressive and potentially fatal. adults).

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